Podcasts about mhra

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a rapidly evolving landscape marked by significant scientific breakthroughs, regulatory shifts, and strategic business maneuvers. The pharmaceutical and biotech sectors are buzzing with renewed vigor, as evidenced by an impressive resurgence in mergers and acquisitions. A recent analysis by PwC reports that M&A activity has reached over $65 billion in deal value during the first quarter of 2026, marking the strongest quarter since 2020. This uptick underscores a robust confidence within the industry, with companies strategically leveraging these mergers to bolster their pipelines and explore new therapeutic territories. Eli Lilly's acquisition of non-opioid pain drugmaker 4E is a case in point, as it reflects a broader industry shift towards precision medicine and non-opioid pain management solutions—a response to growing concerns over opioid addiction. On the regulatory front, notable developments include Colorado's drug import plan receiving FDA approval. This marks a bold step in curbing drug costs across the U.S., although implementation challenges remain due to complex logistical and regulatory landscapes. Meanwhile, Novo Nordisk is expanding its global manufacturing footprint with a new plant in the Czech Republic for diabetes and obesity drugs, alongside a $29 million investment to upgrade its Chinese facility. This expansion aligns with Novo's strategic preparation to seek Chinese regulatory approval for its Wegovy pill, potentially transforming the obesity treatment landscape. In a move that could reshape vaccine development, Moderna is advancing its mRNA-based influenza vaccine candidate through regulatory channels. The FDA's favorable reviews ahead of an advisory committee meeting highlight the growing acceptance of mRNA technology beyond COVID-19 vaccines. This technology holds promise for transforming vaccine development across various infectious diseases. Precision oncology continues to grapple with translating scientific discoveries into practical applications that genuinely improve patient outcomes. The ASCO 2026 conference emphasized this critical transition from discovery to implementation as essential for advancing precision medicine. Turning to gene therapy, UniQure is preparing for a significant milestone—submitting an accelerated Biologics License Application for its Huntington's disease therapy. This follows a reversal by the FDA, which now considers UniQure's Phase 1/2 trial data sufficient for submission. Should this therapy gain approval, it would be groundbreaking as the first genetic medicine for Huntington's disease, setting a precedent for future gene therapies targeting other genetic disorders. In another strategic partnership, Jazz Pharmaceuticals has teamed up with AbCellera to develop T-cell-engaging antibodies for oncology indications, illustrating the potential financial rewards associated with innovative cancer therapies. This collaboration could yield up to $820 million per program and highlights how partnerships are crucial in expediting drug development timelines. These stories reflect broader industry trends emphasizing innovation and strategic partnerships while navigating complex regulatory landscapes. The focus on precision medicine and advanced biologics continues to drive scientific advancements, with companies like Vedana Therapeutics targeting unmet needs in neurology through novel therapeutic approaches. Meanwhile, international collaboration is gaining traction in regulatory processes. The newly launched transatlantic liaison program between the FDA and MHRA aims to accelerate drug approvals and foster innovation across borders—an initiative that underscores the importance of collaborative frameworks. However, not all news is optimistic. Be Biopharma's decision to terminate its hemophilia B cell therapy trial highlights the challenges companies face in competitive therapeutic areas. Despite previous optimism, similar withdrawals by Pfizer and BioMarin indicate the necessity for robust clinical data and clear market differentiation strategies. Furthermore, Merck's recent agreement with Protillion Technologies marks an increased focus on integrating artificial intelligence into drug discovery processes—a trend promising accelerated timelines and improved trial success rates. As these developments unfold, it's evident that the pharmaceutical and biotech sectors are at an intersection where scientific innovation meets strategic business decisions. The potential approval of UniQure's gene therapy could catalyze further advancements in genetic medicine—while M&A activities suggest an industry poised for transformative growth. For stakeholders—from researchers to executives—the ability to adapt to these dynamic changes will be crucial in shaping the future of drug development and patient care. In conclusion, these stories collectively paint a picture of an industry evolving through scientific breakthroughs while adapting through strategic business decisions. As new technologies integrate into this space alongside regulatory advancements in gene therapy, this period of transformation holds promising implications for addressing unmet medical needs and enhancing therapeutic outcomes globally.Support the show

The social media ban for under-16s is officially here - but can government actually enforce it? In Episode 102 of the health and politics podcast, Prevention is the new cure, former Health Ministers Steve Brine and James Bethell dive into Prime Minister Sir Keir Starmer's landmark announcement. From the technical realities of age verification to the political motivations behind the ban, they break down what this means for parents, tech giants like Meta and TikTok, and the future of public health.Plus, a massive game-changer in the obesity crisis: the MHRA has approved the UK's first oral GLP-1 tablet (Wegovy pill) for weight loss. Will a daily tablet shift our public psychology away from "medicalised" injections and into a mass-rollout era? James also reports live from the HLTH conference in Amsterdam on the massive health innovation class divide, what the US-based "MAHA" (Make America Healthy Again) movement gets right about food policy, and why an NHS cybersecurity breach is a matter of clinical patient safety—not just an IT glitch.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a series of significant advancements shaping the landscape of our industry. As technology continues to redefine traditional paradigms, the collaboration between Pfizer and Chai Discovery exemplifies this trend. By harnessing artificial intelligence, particularly through custom models like Chai-3, this partnership aims to revolutionize drug discovery. The integration of AI promises not only to accelerate the identification of biologics and antibodies but also to optimize resource allocation in research and development. Such technological integration could pave the way for an enhanced pipeline of innovative treatments, marking a transformative shift in how therapeutic candidates are developed. In the realm of regulatory developments, Lupin's Ranluspec has recently received FDA approval as an interchangeable biosimilar targeting VEGF-A for various retinal conditions. This move underscores the importance of biosimilars in providing cost-effective alternatives to expensive biologics, thereby expanding patient access to essential treatments for conditions like macular degeneration. Additionally, the MHRA's marketing authorization for Aujemflu, an adjuvanted trivalent influenza vaccine for adults aged 50 and over, reflects ongoing efforts to bolster protection against infectious diseases among vulnerable populations. Clinical trial advancements continue to highlight significant progress in therapeutic development. Otsuka Pharmaceuticals' Phase 3 data on Voyxact has shown promising stabilization of kidney function in patients with Immunoglobulin A nephropathy. This protein therapy targets autoimmune pathways, offering new hope for managing this chronic kidney condition. Similarly, Autobahn Therapeutics' Elunetirom has advanced to a pivotal trial following Phase 2 success in treating bipolar depression. This showcases the potential of small molecule therapies targeting thyroid hormone receptors. Meanwhile, Hikma Pharmaceuticals' victory in a landmark patent case regarding skinny labels marks an important development in pharmaceutical intellectual property rights. The unanimous Supreme Court ruling against Amarin supports the legitimacy of using skinny labels to market generic versions of drugs for non-patented indications. This decision could enhance market competition and drive down healthcare costs, setting a precedent for future intellectual property disputes. On the business front, strategic partnerships and mergers continue to shape industry dynamics. Gilead Sciences' acquisition of Ouro Medicines for $1.675 billion strengthens its autoimmune inflammation pipeline. This transaction exemplifies how major deals are reshaping therapeutic portfolios in response to growing demand for treatments targeting rare diseases. Financially, Solix Pharmaceuticals' success in raising $71 million to advance its siRNA pipeline across multiple therapeutic areas demonstrates investor confidence in RNA-based therapeutics as a promising frontier for innovative treatments. Conversely, challenges persist as evidenced by Takeda's $2.5 billion legal provision over an antitrust case related to Amitiza, underscoring ongoing financial risks associated with litigation in the pharmaceutical sector. Corporate restructuring also signals shifts within the industry landscape. Fulcrum Therapeutics' decision to lay off 85% of its workforce following the discontinuation of its sickle cell disease candidate highlights the volatility and high stakes inherent in drug development. Overall, these developments illustrate a dynamic landscape where scientific innovation is propelled by AI-driven approaches and strategic collaborations while regulatory victories and financial maneuvers shape market dynamics. These trends have profound implications for patient care by potentially accelerating the availability of novel therapies and fostering a competitive environment that drives down costs. As we look ahead, stakeholders must navigate these complexities effectively to harness opportunities and address challenges within this rapidly evolving industry landscape. The ability to adapt and capitalize on emerging trends will be crucial as these sectors continue to evolve, ultimately enhancing patient care and advancing therapeutic frontiers globally. Thank you for joining us today on Pharma Daily; stay tuned for more insights into the ever-changing world of pharmaceuticals and biotech.Support the show

Misha Glenny and guests discuss one of the great writers on Central Europe after the first world war and on the dying of the old orders with the collapse of the Austro-Hungarian empire. As a German speaking Jew from Brody in the north-eastern edge of that Empire, which was then in Galicia, next in Poland and is now in Ukraine, Roth (1894 - 1939) was to spend his short life moving first to Lviv then to Vienna and finally to Paris via Berlin without ever finding a settled home. Roth explored the loss of homeland and anticipated the dangers of the new nationalism through his journalism and in his novels including Radetzky March, Job, Rebellion and Flight Without End, and his books were among the first the Nazis burned.With Helen Chambers Emeritus Professor of German at the University of St AndrewsDeborah Holmes Associate Professor of Modern German Literature at the University of SalzburgAnd Jon Hughes Reader in German and Cultural Studies at Royal Holloway, University of LondonProducer: Simon TillotsonReading list:Jon Hughes, Facing Modernity: Fragmentation, Culture and Identity in Joseph Roth's Writing in the 1920s (MHRA, 2006) Heinz Lunzer and Victoria Lunzer-Talos, Joseph Roth: Leben und Werk in Bildern (Kiepenheuer & Witsch, 1994)Keiron Pim, Endless Flight: The Life of Joseph Roth (Granta, 2022)Joseph Roth (trans. Deborah Holmes, ed. Helen Constantine), Vienna Tales (Oxford University Press, 2014)Joseph Roth (trans. and ed. Michael Hofmann), A Life in Letters (Granta, 2012)Joseph Roth (trans. Michael Hofmann), Collected Shorter Fiction (Granta, 2001)Joseph Roth (trans. Michael Hofmann), Rebellion (Granta, 2000)Joseph Roth (trans. Michael Hofmann), The Radetzky March (Granta, 2022)Joseph Roth (trans. Michael Hofmann), The Legend of the Holy Drinker (Granta, 2022)Joseph Roth (trans. Michael Hofmann), The Wandering Jews (Granta, 2001)Joseph Roth (trans. Michael Hofmann), What I Saw: Reports from Berlin 1920-1933 (Granta, 2022)Joseph Roth (trans. Michael Hofmann), The Hotel Years: Wanderings in Europe Between the Wars (Granta, 2015)Joseph Roth (trans. Michael Hofmann), Reports from a Parisian Paradise: Essays from France 1925-1939 (Granta, 2004)Joseph Roth (trans. Michael Hofmann), The Emperor's Tomb (Granta, 2013)Joseph Roth (trans. Michael Hofmann), The String of Pearls (Granta, 1999)Joseph Roth (trans. Michael Hofmann), The White Cities: Reports From France 1925-1939 (Granta, 2013)Joseph Roth (trans. David Le Vay), Weights and Measures (Pushkin Press, 2024)Joseph Roth (trans. Daved Le Vay and Beatrice Musgrave), Flight Without End (Pushkin Press, 2024)Joseph Roth (trans. Ruth Martin), The Coral Merchant: Essential Stories (Pushkin Press, 2020)Joseph Roth (trans Will Stone), On the End of the World (Pushkin Press, 2019)Joseph Roth (trans. Dorothy Thompson), Job: The Story of a Simple Man (Granta, 2022)Wilhelm Von Sternburg, Joseph Roth: Eine Biographie (Kiepenheuer & Witsch, 2009)In Our Time is a BBC Studios ProductionSpanning history, religion, culture, science and philosophy, In Our Time from BBC Radio 4 is essential listening for the intellectually curious. In each episode, host Misha Glenny and expert guests explore the characters, events and discoveries that have shaped our world.

Artificial intelligence is developing at breakneck speed, leaving governments around the world scrambling to respond. For a high-risk area like health care, safe, responsible use will be critical. But pressures on health systems mean governments can ill afford to delay adoption. So what's the right balance? And how can we ensure AI tools work in real-life health care settings and minimise unintended consequences?Following the 10-Year Health Plan, the government has established a National Commission into the Regulation of AI in Healthcare to explore these questions and make recommendations for how regulations need to adapt. To discuss, our Chief Executive Jennifer Dixon is joined by: Ricardo Baptista Leite, CEO of Health AI, a Geneva-based nonprofit that promotes equitable access to AI-powered health innovations. Alastair Denniston, Professor of Regulatory Science and Innovation at the University of Birmingham.  The National Commission into the Regulation of AI in Healthcare is established to advise the MHRA. It is co-chaired by Alastair Denniston. Ricardo Baptista Leite and Jennifer Dixon are members. Its final report is expected later in 2026. Show notesUK government. National AI Commission into the Regulation of AI in Healthcare.Health Foundation (2025). AI in healthcare – staying ahead of the issues. Health AI (2026). AI governance in health – Global landscape 2025 report.  Health Foundation (2026). AI in healthcare must earn the public's confidence. JAMA (2025). AI, Health, and Health Care Today and Tomorrow: the JAMA Summit Report on Artificial Intelligence.

James joins from the Milken Institute Global Conference in Beverly Hills this week and the guys unpack five "states of mind" currently dominating the global health and politics landscape. From China's surging dominance in biopharma innovation to the sobering reality of the failed resource shift toward prevention, this episode is a wake-up call for Western policy makers.In this episode, we discuss:The China Reckoning: How Jiangsu Hengrui overtook AstraZeneca and what it means for UK/US national security.The AI "Crash": Why 90% of AI startups may fail and what the "Middle of AI" looks like.The MHRA's New Vision: Insights from Lawrence Tallon on digital technology and international reciprocity.US Politics & Health: Will Dr. Oz replace RFK Jr. in the American health landscape?The "Pre-loading" Debate: Should airport alcohol sales be banned?Connect with us:

With a lot in the news at the moment about the possible side affects of some medicines we thought that for this week's check in with Glasgow based Pharmacist Elizabeth Roddick we should talk about some of those potential side effects and what happens if a medicine needs to be recalled.Elizabeth began by explaining to RNIB Connect Radio's Toby Davey about the potential issues of the overdose of vitamin D in children. Then to what happens if there was a faulty product such as a supplement and how that is dealt with differently to what happens when there is a problem with a medicine.To then looking at what happens when a medicine needs to be recalled and the Yellow Card System within the Pharmacy that informs the MHRA if there is a problem with a medicine.To get in touch with Elizabeth you can email info@elizabethroddick.com or for more information visit - https://elizabethroddick.comImage: Picture showing Elizabeth standing in her pharmacy, dressed in her white pharmacist coat and a colourful bandana around her neck, she's smiling warmly at the camera with her hands slightly raised.

hVIVO PLC (AIM:HVO) chief executive Yamin ‘Mo' Khan talked with Proactive's Stephen Gunnion about the company securing a landmark contract to deliver the world's first pivotal Phase III human challenge trial for ILiAD's whooping cough vaccine candidate. Khan explained that the agreement marks a major milestone for hVIVO, representing the largest contract in the company's history and involving the highest number of clinical participants it has ever managed. He highlighted the broader implications for patients, noting that human challenge trials can significantly accelerate the path to market for new treatments. “This has three major impacts,” Khan said, pointing to faster patient access, a transformative contract for hVIVO PLC, and a precedent-setting moment for the wider clinical trial industry. The study will use a human challenge model to assess vaccine efficacy in a controlled environment, addressing the unpredictability of whooping cough infection rates in traditional trials. Khan noted that regulators, including the FDA, MHRA and EMA, have aligned on the approach, enabling faster and more cost-effective development. He also emphasised hVIVO's unique positioning in the sector, stating, “We are the world leader when it comes to human challenge trials,” citing its experience conducting over 50 trials and inoculating more than 5,000 participants. The trial is expected to begin this year, with revenue contributions forecast across 2026 and 2027, enhancing both short- and mid-term visibility for the company. For more videos like this, visit Proactive's YouTube channel, give this video a like, subscribe to the channel and enable notifications for future content. #hVIVO #Biotech #ClinicalTrials #VaccineDevelopment #WhoopingCough #Phase3Trial #HumanChallengeTrial #ILiAD #PharmaNews #HealthcareInnovation #BiotechInvesting #LifeSciences

Andrew Bridgen, former UK MP and tireless campaigner for accountability, discusses his ongoing legal battle against Matt Hancock (former UK Health Secretary) over defamatory statements made during the COVID era, the suppression of vaccine injury data, and the shocking MHRA survey revealing that 1 in 7 participants experienced a severe adverse event from the COVID vaccines.He covers the deliberate destruction of UK energy security through net zero policies, the weaponisation of Lawfare against farmers and dissenters, and the failure of legacy media and institutions to protect the public. With elections approaching and public trust in government and media at rock bottom, Andrew calls for genuine opposition, transparency, and resistance to the uni-party system.Andrew Bridgen Meets Vaccine-Injured Actor & Writer Barry Duffield (2026): https://rumble.com/v73zkia-andrew-bridgen-meets-vaccine-injured-actor-barry-duffield.html?e9s=src_v1_cbl%2Csrc_v1_ucp_aAndrew Bridgen on X/Twitter - https://x.com/ABridgenAndrew Bridgen's website - https://abridgen.uk/fund-my-work/Support FreeNZ:Substack: https://freenz.substack.com/Locals: https://freenz.locals.comBuy Me A Coffee: www.buymeacoffee.com/supportfreenzKo-fi: https://ko-fi.com/freenzhttps://freenz.carrd.coAffiliates:Dioxi Care - Chlorine Dioxide based Oral Care, Skin Care & Veterinary & Wound Care: https://frontierpharm.com/?sca_ref=9717384.brQladA5pgSnoot Spray - Chlorine Dioxide based Nasal Cleaner: https://www.snootspray.com/?sca_ref=9667634.AV2NJQvGlTNavigate the matrix with this 2026 Year of the Fire Horse Numerology & Astrology Calendar: https://gumroad.com/a/809846675/itcfmgWide Awake Media - Freedom T-Shirts: https://wideawake.clothing/en-nz?sca_ref=9458851.1aXfjvGDqL

A flurry of deals, several with sizable upfront payments, has energized the biotech sector in March. On the latest BioCentury This Week podcast, BioCentury's Paul Bonanos assesses last week's deals by Merck, which is laying out $6.7 billion to acquire Terns, and Gilead, which is buying Ouro for about $1.7 billion up front.Gilead's deal comes as the Foster City, Calif.-based biotech is looking to defend its position in HIV while resetting in inflammation and immunology and growing its oncology footprint through business development. BioCentury's Lauren Martz analyzes the company's pipeline following her recent conversation with CMO Dietmar Berger.Turning to the U.K., Editor in Chief Simone Fishburn details her conversation with CEO of MHRA, Lawrence Tallon, regarding what new reforms in the U.K. mean for drug developers and how he views initiatives helping to build the sector and place the U.K. in the race to become a destination for clinical trials.Rates for the 26th Bio€quity Europe May 4-6 in Prague increase after this week. Register now as a delegate or apply to join the 2026 Presenting Company Class before the conference sells out.View full story: https://www.biocentury.com/article/658974#BiotechMA #BiopharmaStrategy #DrugDevelopment #UKBiotech #ClinicalTrials00:00 - Introduction01:09 - Merck Buying Terns04:31 - Gilead, Galapagos Deal09:35 - Growing Gilead19:20 - Tallon's Vision for U.K. BiotechTo submit a question to BioCentury's editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

In this episode, we explore how individualised medicines are evolving from “n=1” treatments (a treatment effective for a single individual) into approaches that could transform care for many people living with rare conditions.  Advances in genomic medicine are making it possible to design highly targeted treatments based on an individual's genetic information. While these therapies may begin as bespoke solutions for a single patient, they can often be adapted, refined or reused to benefit others with similar conditions.  While the research is evolving, the systems needed to deliver these treatments at scale are still catching up. From regulation to access, our guests discuss what needs to change to turn this potential into reality.  Our host Sharon Jones, is joined by:  Ana Lisa Tavares, Clinical Lead for Rare Disease Research at Genomics England  Mel Dixon, Participant Panel member and CEO and Founder of Cure DHDDS  If you enjoyed today's conversation, please like and share wherever you listen to your podcasts.  “However rare your condition is, someone has a right to have hope. Everybody should have a hope that we should be able to find a treatment.” You can download the transcript or read it below. Sharon: What if treatments once designed for just one person could now help many others? Thanks to advances in genomic medicine, regulations are changing and research is expanding. This opens up more options for treatments for rare conditions. But what does this mean and how close is real change? I'm Sharon Jones, and this is Behind the Genes. We look at how genomics is changing healthcare, covering everything from cutting-edge research to real-life stories. Individualised medicines are a fast-moving area, but there's still a big gap between scientific progress and what's actually happening to patients. You could call it the gap between hype and hope. Ana Lisa: However rare your condition is, someone has a right to have hope. Everybody should have a hope that we should be able to find a treatment. Sharon: Coming up, we'll hear from Ana Lisa Tavares, Clinical Lead for Rare Disease Research at Genomics England, and Consultant in Clinical Genetics at Cambridge University Hospital, as well as Mel Dixon, member of the Participant Panel at Genomics England and CEO and founder of Cure DHDDS. Mel opens this chat by explaining why developments in individualised healthcare really matter to her. Mel: This issue is really personal to me. I have three children, two of whom are affected with an ultra-rare DHDDS gene variant, for which there is currently no treatment. Their condition causes symptoms such as, well, it varies between mild to severe learning difficulties, seizures, tremors, and movement and coordination difficulties. But the, the most worrying thing for us was that this condition is actually also progressive. So over time it becomes more of a Parkinsonism and some patients experience dementia-like symptoms and psychosis. So for us to get a treatment that targets the genetic cause of, of their condition is, like, the most important thing in, in our lives. If we could intervene now, they could potentially, at the stage they're at, you know, live an independent life with, with some supports. But if the disease is left to progress, it would be a very different outcome for them. Sharon: I mean, that sounds so difficult and I can't even imagine how life is for you and your family. And I can see what is driving you to find anything to extend the life of your children and to give them that opportunity to, to have a better quality of life. And then Lisa. Ana Lisa: It's a huge burden for families to carry. And I think at the moment there's an additional layer of burden, which shouldn't fall on families, to feel like they need to forge a pathway for their child to have a chance of a treatment. That's, that's a lot to bear. Mel: I think as well, families feel they almost have to become mini scientists in their children's specific condition overnight, because you go to these appointments with the consultants and nobody's heard of the condition and they don't know, they just don't really know what to do with you. So they're asking you, you know, so tell me about this, this gene change. What, what does it do? What does it mean? So you have to become the mini professor in your child's condition to be able to advocate for them. We've had to really learn on our feet so that we're able to advocate and push for research into DHDDS, because without us doing it, nobody else was going to be. Sharon: Yeah. So that's, you know, that's partly what we're here and what this podcast is for, it's here to support families to, to understand this stuff. And Ana Lisa, can you just break it down to us, what is individualised medicines? Ana Lisa: An individualised medicine that's made for one individual person. In reality, sometimes there are other individuals that can also benefit from the same medicines, and sometimes actually, although the medicine is made for one specific person, it might be made using a strategy that other patients could also benefit from, either directly, exactly the same, even, or through tweaking them so that they could work for a different patient. In the context that they're most often referred to at the moment, they're therapies that are being made based on the genetic information about somebody. Sharon: Thank you. I mean, that sounds amazing. And now coming to you, Mel, what does receiving a diagnosis mean for a family? And how do you navigate the space between finally having answers and the reality that the treatment may not yet exist? Mel: So for us, I think, we went down the, the diagnostic route in the hope that we would be able to find a treatment for our children, or there would already be a treatment in place. But unfortunately when we got their diagnosis, we were told that their, their condition was ultra rare, neurodegenerative and also newly discovered. So there was, there was no treatment pathway and actually minimal research happening into it at the time. So it was frustrating, upsetting, um, and it felt like quite a hopeless situation at the start, but actually this was just over three years ago. And through a lot of proactiveness on our part in fundraising, we've been able to better understand the condition and we now have treatments in the pipeline. So in that three-year window, from there being nothing, we now have treatments both in terms of potential drug repurposing candidates and also, um, an individualised therapy called an ASO is also in development for them. So it was hard, but it's given huge benefit to us. Otherwise, we'd just be going, remaining going from specialist to specialist without having any answers or understanding why their symptoms were progressing. Sharon: I mean, that sounds really, really tough and you know, coming back to you, Ana Lisa, could you talk us through how genomics is changing the way we can treat rare conditions? You know, what types of individualised medicines now exist and how do they even work? Ana Lisa: Maybe I'll start with how some of these medicines are working. So with, without going into details, but the sort of principle that these medicines might be able to, to do something called gene editing. So our, our DNA, uh, the instruction manual is made up of genes and it's now can be possible scientifically to change even a single DNA letter code in somebody to try and ameliorate the symptoms of their rare condition. You know that's phenomenal scientific progress to be able to do that. I think a lot of people have heard about gene therapy, where one is trying to get into the body a gene or part of a gene that might be able to sort of replace the function of a gene that isn't working as it should. There are various other strategies. So our DNA is actually used to send messages to our body, if you like, to, to decode these instructions. And so there are medicines that target the next step in this process, the RNA, which are the ASO therapies that Mel was referring to earlier. And really what those are doing are either trying to correct for a protein in our body that isn't working as it should, or to try and get rid of one that shouldn't be there. And so they can act in different ways. And that's actually quite powerful, because you can, theoretically, use these strategies to correct for different genetic rare conditions. So I think going to the sort of first part of your question, maybe if I can phrase it as "directly at source". If you can go upstream and target in a very direct way the cause of a rare condition, then actually you might be able to apply those same principles to many different types of rare condition. We know that there are, you know, 8,000 as a very ballpark number of rare conditions, and it might be that these strategies could be used I don't want to say for all rare conditions, but for many rare conditions where we find the genetic cause, these strategies could collectively be a very powerful way to treat them. And traditionally we've had to understand all the underlying biology, find a druggable target, find a drug that could target that, that's safe, effective, et cetera. And that's a lot of work. And that's still very, very valuable. If we were going to do this for these thousands of conditions, it would probably take us hundreds to thousands of years, collectively. And these strategies provide a lot of hope for being able to do this in a, in a more efficient way, where we can actually use the information used to treat one rare condition and apply those learnings to another rare condition. Sharon : I mean, that's really helpful to understand. So if the science is there, why aren't more patients benefiting from it yet? You know, what's standing in the way from your perspective? Ana Lisa: That's a really good question, and it's complex because the, our whole ecosystem is made up of, of many parts that go from finding a potential strategy that could help a rare condition to a patient benefiting from that. And I think one thing that maybe we haven't touched on yet is the fact that rare conditions can be really rare and affect a really small number of people individually, even though we know collectively they affect so many. You know, in the past it's been easier, if you're taking a condition that's common, that affects thousands of people, it's easier to see and to be sure whether your new medicine is actually working as you think it does and should, and having the benefits that you think. The, the sort of regulators have really clear guidance. We have lots of knowledge about how to assess treatments and have a randomised clinical trial, for example. How the reimbursement process may work in a public healthcare system. And when you, when you, when you sort of set down into the really rare, this is difficult for each stage of the journey. The transformation that's needed is a whole, system-wide transformation to be able to regulate in a scalable, equitable way, these therapies that could actually be an N of one treatment for one individual, that actually maybe one day another individual may also benefit, and sometimes even a group of individuals. It's not just the, the regulator, it's also how do you make it viable. So again, you have to make it scalable, equitable. And even to implement in the NHS down to this very "N equals one" level, and demonstrate that patients could benefit from these treatments, might require sort of fancier ways of assessing these treatments, whether it's statistics, other methodology and I think it's really the system-wide nature that makes this tricky, but is also a fantastic opportunity for, for collaboration, because that, that sort of end goal and benefits could be so, so great. Sharon: Yeah, absolutely. And I mean, Mel, for your side of things, it must sound, you know, quite frustrating where the people in the rare community to not see the support being made more readily available? Mel: Yeah, it is particularly difficult for patients and their families. I think in our case, when you're dealing with a neurodegenerative condition, time is of the essence. So when you know that the science is available or it's ready, but you don't have the systems in place to implement them to the patients so that they can access these much-needed therapies, it's worrying and frustrating. And also I see our children are affected with, with, you know, one of these N of few conditions that there's, you know, there's only 59 confirmed cases of DHDDS worldwide, and we've seen how the system firsthand doesn't fit ultra-rare patients. We can't, when we were looking at drug repurposing, we can't do a traditional clinical trial because we don't have the patient numbers and we don't have the funding. So a placebo-controlled trial just wouldn't be possible for us when there's only, I think, seven confirmed patients in the UK and, um, four that we're actually in, in, in touch with. So it does feel, I think, as Ana Lisa was saying, that we really need a system rethink, um, and refit so that it does start to accommodate these ultra-rare conditions, especially now as there's therapies which are showing huge benefit to patients. Sharon: And so with like all of these challenges, where are you seeing things shift and what does meaningful progress really look like for you? Mel: At the end of last year, the MHRA announced that they were rewriting the regulatory framework for rare conditions, and that fills us with lots of hope for the future. They're recognising that the traditional systems don't work for particularly ultra-rare conditions, and now that we do have these therapies in the pipeline, we, we want to get the patients to be able to access them. And we're also seeing innovation in how evidence is generated and measured. We witnessed this firsthand with our son as he was undergoing baseline tests for his ASO therapy. You know, the use of digital biomarkers, of real-world evidence, how they're increasingly being used for these N of one or N of few populations. And how the individual receiving the treatment becomes their own comparator. So you're not relying on these big natural history studies of the disease or placebo controls. It's you're looking specifically at that individual, getting a really strong baseline and then looking, once they're dosed with the medication, is that improving or stabilising symptoms? So I think this shift in focus is really meaningful for the ultra-rare community and also for them to be part of the decision-making process of what, what benefits do they want from a drug? Like what is meaningful to them? I think there's much more talk about the patients and how the, what will benefit them most. It's not necessarily what the scientists would think or research would think would most benefit, but what, what would make a meaningful difference to the patient? Sharon: I mean, that's good to know because it's kind of putting the person at the centre of, you know, this is what it's all about, isn't it? It's not just the science. We're trying to treat people and it's putting people, people first. Ana Lisa: Just to build on that, it's exactly that, that awareness that is, is growing, I think, that there are so many people affected by a rare condition and, and however rare your condition is, someone has a right to have hope and that the system should be able to cater for many rare conditions, you know, whether they're an ultra-rare or an actually almost common rare condition, everybody should have a hope that we should be able to find a treatment. And it's not a hopeless situation that it's, you know, never going to happen or be too difficult. It's quite powerful, hope. If you can solve for the truly individualised medicine, then you at the same time may also be helping everyone in-between a really common condition and a really rare condition, because right now the system works for common conditions. And if you can take it right down to the sort of radical of, example of an individualised medicine made for one person, then you are also forcing the system to a change for everybody else. And I think that's one of the great benefits of thinking about it as a joined-up system. Sharon: So how do you each navigate between hype versus hope when it comes to rare therapies? Mel? Mel: I like to focus on hope, because when we got our diagnosis, we felt really hopeless and that's a really dark place for a family to be. But as we learnt more about their condition and the rare condition landscape and genomics, we actually learned of all these new therapies that were in the pipeline. We were hearing about, you know, recently, conditions like Huntington's Disease that you never, never previously had any disease-modifying treatment, how they're now being able to be treated with gene therapy with really positive effects. Similarly for other neurodegenerative conditions that have been treated with ASOs, how they're seeing not just disease stabilisation, but improvements. So I know it's, it's still, like, relatively early days with these technologies and therapies, but I think it, it allows families to have hope, which is, which is really, really important, because that statistic, you know, of the, of 95% of rare conditions not having a treatment, it's, it's a really brutal one, uh, to be told at the outset or to learn at the outset. So, you know, if, if these therapies can, can make a huge dent in that, that would be life-changing. It would make a profound difference to many, many families, and I think there's a lot of reason to have hope, taking all of that into consideration. Sharon: And then Lisa? Ana Lisa: I think to work in this area, one needs to be full of hope and optimism because there are so many, um, challenges to overcome as a community. Uh, but I think that means that people are also incredibly collaborative, because they know that we need to work together for this to succeed. And no one, you know, one individual, one organisation can do it on their own. It truly has to be a crosscutting, collaborative endeavour. The fact that we, in the UK, have resources like the National Healthcare System,Genomics England in partnership with the NHS runs a National Genomic Research Library. And so the fact that you could look at, at tens of thousands of, of genomes for many, many individuals with rare conditions. That gives me hope because it means that if a treatment is made for another person, it could be in a different country in the world, and if we could find another patient, it doesn't matter what specialty they're under, where they are, we should be able to find them and connect with their clinical team if, you know, if they've consented for the National Genomic Research Library. And so to me, that feels, that whilst there's, there is a lot of hype in the sense that some of the really well-publicised cases, really had a lot of people working on them and a lot of resources to make it happen. But that gives hope to everybody else that follows that actually it is doable and if we can make better systems, and having these national resources that we do, the fact that, there are a lot of guidelines being written at the moment, both international and national. And again, they show that the sort of scaffolding is starting to be in place to apply these in an equitable scalable way. It might not be that you're so much looking for a specific rare condition as for a particular type of genetic variant that could be targeted in the same strategic way, and that therefore you could look across many different rare conditions. So again, all these sort of pieces of the puzzle are, are filling me with, with, with hope. Sharon: You touched upon, um, inequity there. Now, you know, is there a risk of inequity given what we've talked about in terms of those challenges? Ana Lisa: I think we, we always have to have the lens of equity in everything we, we do. And that, and that really does apply to healthcare and, and in fact, probably the whole rare disease community are, are, are not well served in terms of therapies at the moment. There are so few, um, therapeutic options and so I think there's a massive inequity in that this, our systems are not geared, uh, towards rare conditions. I suppose, you know, different countries have different healthcare systems and some of the sort of first personalised therapies may require a lot of money behind them to, to happen, but they will be pioneers in leading the way for how this can be done. And I think in the UK we have a lot of the infrastructure and the, a sort of a strong, that's very equitable, I think. And so we could do this in a, in a much more open and equitable way. Sharon: Mel? Mel: Cost is always, unfortunately, and it, when it's your family that's affected you, you know, you hate the thought that things are coming down to cost and, and money. But I, I think as Ana Lisa said, if, if the system absorbs the initial cost. You know, it seems that those longer-term costs could come down significantly. We already see with our very small DHDDS community that an ASO, which is an allele specific that was made personally for one, for one child, can actually also benefit my son, even though they have a different variant. So if the cost of the ASO is 1.2 million per person, but if you suddenly find actually one other person can share that, that's almost halving the, the cost. And then if then you're finding out that actually, oh no, 3, 4, 5, 10 people can all have that same ASO, suddenly it becomes much more cost-effective and more sustainable. So I think, as we have to think about cost, I think that also allows us to have more hope that these therapies can, the cost of these therapies that are obviously hugely expensive at the moment, can be brought down in the longer term. Ana Lisa: There are a lot of things that people want to do in the NHS. People can be working under quite hard circumstances, so to talk about making a therapy for one individual can be difficult and people can sometimes, I think, think that it's a pie-in-the-sky conversation. However, I think that, you know, all the clinicians I know who work with families with rare conditions, what they'd most like to be able to do is to be able to offer a therapy. And so I think a lot of people see this as a, as a big opportunity, despite these initial hurdles. One thing I often think about is my grandfather, when he was alive, every phone conversation, he would start with, "How many lives have you saved today?" And so I think that's the, that's our challenge. Sharon: Wow. That's, that's really powerful. Mel: Just echoing really what Ana Lisa was saying, I feel the, um, inequity lies in rare conditions as a, as a whole, from the point of diagnosis to the lack of pathway, um, to, to the lack of system in place for them. You wouldn't have a patient with a life-changing cancer diagnosis receive that information in a telephone call, and that is the stark reality for many rare disease patients. That's how they receive the, that's how they often receive the news. That was certainly our, our experience. And, and from that point, there was then no pathway. It's just this horrendous feeling of isolation. And I think now that there are these treatments in place and therapies in, in place, it's about time we change that because often the rare, the rare condition community, and certainly those with ultra-rare conditions as well, they're probably like some of the most underserved members of the community in that it's their parents and their families that have to advocate. Otherwise, without that, they, they often wouldn't stand a chance of understanding the disease, let alone finding a treatment. So I think the whole system needs to have a reset, to think about these rare condition patients and, put them at the heart as they do for more common conditions. Ana Lisa : I completely agree. And you mentioned cancer, and there are actually quite a few parallels. So there might be really common cancers that affect a lot of people that are being, uh, subsetted down into different groups depending on the genetics that are related to that particular cancer and therefore what treatments might be most effective. And so I think there's, there's a lot we can each learn from each other between the rare disease and cancer communities. Perhaps as in rare disease we scale up to apply the same strategies to many different rare conditions and patients. Even if they're being tweaked for their particular genetic variant and cancer, sometimes one is subsetting down to treat specifically that, exactly that cancer subtype. So there's a lot we can learn and I completely agree that the, the rare disease community deserves the same chance at at treatments, and the hope that that comes with. Sharon: Thank you. It feels like there needs to be some kind of seismic system change along with this piece around collaboration and how, you know, the science is there, but it's how do we bring it to families who are facing these difficulties with it, you know, their children and, and rare conditions. We'll wrap it up there. Thank you to our guests, Ana Lisa Tavares and Mel Dixon, for joining me as we discussed the evolving landscape of individualised medicines. And thank you for listening. If you'd like to hear more like this, please subscribe on your favorite podcast app. Behind the Genes is produced by Deanna Barac, Florence Cornish, Sophie McLachlan and Patrick Wallace at Bespoken Media.  

Note: This was recorded on 26th February 2026. We apologise for the late release.UKMFA directors Ian Humphreys and Dr Liz Evans with a lively and wide-ranging conversation. Reporting on recent UKMFA work, and stories from the news and personal anecdotes which raise concern, from a medical ethics perspective.Topics we discussed included:UKMFA Director Dr Sarah Myhill's two recent FOI requests to the GMC Chair and CEO and Prof Chris Whitty.The GMC Chair and CEO have been FOIed for documents discussing; the need for full informed consent for every Covid jab, supporting individual doctors punished for resisting political or employer pressure to circumvent informed consent to vaccination. And all communications with the UK Government regarding the Covid jab rollout.Chris Whitty has been FOIed to provide the evidence base that he relied on during the Covid vaccine rollout to support his claims that the jabs were "Safe and Effective". And for the raw data from the MHRA and Yellow Card System reporting Covid vaccine harms.UKMFA Supporting Whistleblower Doctors - with a Joint Open Letter to the GMC to Support Dr Aseem Malhotra and recent UKMFA interview with Dr David Cartland “Treated Like a Criminal”.BREAKING NEWS - Today we published a Joint Open Letter to the DBS and FA in support of Dr Cartland, signed by nearly 80 doctors, academics and healthcare professionals.News of ex-MHRA Chief Dr Ian Hudson, struck off the medical register this month by the GMC for failing to report child sex offence convictions in 2024.Acceleration of NHS Digital 10 Year Plan - with the introduction of AI receptionists, destruction of the doctor-patient relationship, and serious access problems for patients to see a doctor in person.NHS Push to Prevention away from Treatment - GPs being incentivised to speed up the weight-loss jab rollout and give MORE vaccines. Meanwhile, healthy life expectancy is falling rapidly despite NHS spending doubling over the last 17 years.Please email personal stories or documentation/letters relating to medical ethics breaches to us at podcast@ukmedfreedom.org. In particular on:Increasing barriers to face-to-face access your GP, especially if it led to harm.Pressure from the NHS or Vaccination UK on parents for their children to participate in school vaccination days. And incidences where children have been vaccinated without or against documented parental consent.UKMFA:CALL TO ACTION: Please follow us and subscribe on our YouTube and Rumble channels and please share our content on social media and with friends and family, to help us get the message out and increase our reach.All our podcasts can also be found on the major audio platforms e.g. Apple and Spotify.Our new Substack is found here: https://substack.com/@ukmfa1We are grateful for all donations to help us to continue and grow our work; lobbying decision makers; educating and empowering the public; running campaigns and producing our podcasts. On screen you will see a QR code which please scan using your mobile device. You can always use this link to donate directly: https://donorbox.org/ukmfa_p

On this fortnight's episode, our embattled hosts Flint, Ashleigh & Alyx go over: Difficult news from the Southern states of the US in Pond Hopping. CONTENT WARNING: This story contains talking about suicide, potential murder and racism. This is signposted within the episode and a time code to skip over this story is included. TransActual's new report "Continuing to Endure the UK's Hostile Environment. How a particular professer is prohibited from participating in the PATHWAYS trial by the MHRA. Our main section talks about the now 'official' halting of HRT prescriptions to 16 and 17-year olds.  References: https://whatthetrans.com/ep151 Submit to a zine! Extended deadline: March 28th - Open Call — Carnations, Violets & Lavender

MedboardSponsor: Medboard: https://www.medboard.com/EuropePharmacist are distributors - French guide issued to educate them: https://medboard-public-assets.s3.amazonaws.com/Regulatory/9b892d08-611c-4326-a40e-750e0364df27.pdfTeam NBTeam NB position Paper - Reduced Scrutiny, Cost saving: https://www.team-nb.org/wp-content/uploads/2026/03/Team-NB-PositionPaper-MDR-IVDR-revision-impact-on-the-sector-20260302.pdfTeam-NB Demonstrate Safety and performance - For Combination of reagent and other equipment: https://www.team-nb.org/wp-content/uploads/2026/02/Team-NB-PositionPaper-Demonstration-of-Safety-and-Performance-for-Combinatorial-Use-of-Devices-or-Equipment-V1-20260216.pdfTeam-NB- Letter on Cybersecurity - New Proposal again: https://www.team-nb.org/wp-content/uploads/2026/02/Team-NB-Letter-on-cybersecurity-20260205.pdfTeam-NB - MDR Clinical & Tech File Training - Check the dates: https://medboard-public-assets.s3.amazonaws.com/Regulatory/442102-Leaflet-MDR-Clinical-Manufacturers-Training-20260528.pdf   and   https://www.team-nb.org/wp-content/uploads/2026/02/Leaflet-MDR-TD-Manufacturers-Training-20260429.pdfUKConsultation on recognizing CE mark - Should it be indefinitely: https://www.gov.uk/government/consultations/medical-devices-regulations-targeted-consultation-on-the-indefinite-recognition-of-ce-marked-devicesConsultation until: April 10, 2026 UK Reliance is ongoing - Draft proposal to recognize MDSAP and other: https://www.gov.uk/government/publications/implementation-of-the-future-regulation-of-medical-devices/statement-of-policy-intent-international-recognition-of-medical-devicesBookmark the MHRA contact page -*Whispering* If you want to Whistleblow, there is a contact Chuuut!!!: https://www.gov.uk/guidance/contact-mhraSolutionsEasyIFU: For eifu and Label -  Test it for Free: https://easyifu.comSmarteye: for eQMS . Ask for a Demo: https://eqms-smarteye.com/RoWIMDRFReliance Playbook - With examples to apply: https://www.imdrf.org/sites/default/files/2026-02/IMDRF%20GRRP%20WG%20N89%20Reliance%20Playbook.pdfUSAFDA Cybersecurity documentation - Final Guidance: https://www.fda.gov/media/119933/downloadMalaysiaLaunch of the Innovative Pathway - Accelerate the development of your device: https://portal.mda.gov.my/index.php/announcement/1771-implementation-of-the-innovative-medical-device-review-pathway-by-medical-device-authority-ministry-of-health-malaysiaArgentinaSelf Declaration expended for Low-Risk devices - Themis platform to be used: https://www.argentina.gob.ar/noticias/entra-en-vigencia-el-nuevo-regimen-simplificado-para-la-habilitacion-de-establecimientos PodcastEpisode 376 - Defect Management in SaMD with Anindya Mookerjea: https://podcast.easymedicaldevice.com/376-2/Episode 377 - IEC 60601 4th edition with Leo Eisner: https://podcast.easymedicaldevice.com/377-2/Episode 378 - IMDRF & Regulatory Reliance with Stephanie Grassmann: https://podcast.easymedicaldevice.com/378-2/Social Media to followMonir El Azzouzi Linkedin: https://linkedin.com/in/melazzouziTwitter: https://twitter.com/elazzouzimPinterest: https://www.pinterest.com/easymedicaldeviceInstagram: https://www.instagram.com/easymedicaldeviceThis podcast is powered by Podcastics, the easiest platform to create and publish your podcast.

In this episode of Data in Biotech, host Ross Katz sits down with Ben Locwin, Vice President at Reliant Life Sciences, to explore the evolving landscape of artificial intelligence in biotechnology.  Join us as we discuss why nearly every biotech claims to use AI but few actually do, examine successful applications like AlphaFold, and explore the challenges of implementing AI across drug development, manufacturing, and regulatory processes. Ben shares insights on maintaining healthy skepticism, understanding data provenance, and looking ahead to what this year may bring for AI in life sciences. What you'll learn in this episode:  >> The AI hype problem in biotech and why most companies claim to use AI but few actually do. >> AlphaFold as the gold standard and how DeepMind's protein structure prediction model represents the most successful application of AI in biotech >> Data quality over algorithmic sophistication and the critical importance of data provenance, examining primary sources, and understanding that data quality matters more than the complexity of the AI model >> The balance between optimism and evidence-based decision-making, distinguishing between sophisticated AI and advanced statistical modeling Meet our guest: Ben Locwin is a healthcare and life sciences executive and medical scientist known for helping bring pharmaceuticals, vaccines, and medical devices to market faster and with higher quality. A TEDx speaker and seasoned leader, he's worked across major biotech hubs and has deep expertise in global regulatory pathways, having collaborated with the FDA, EMA, MHRA, PMDA, and more. Connect with Ben Locwin on LinkedIn  About the host: Ross Katz is Principal and Data Science Lead at CorrDyn. Ross specializes in building intelligent data systems that empower biotech and healthcare organizations to extract insights and drive innovation. Connect with Ross Katz on LinkedIn Connect with us: Follow the podcast for more insightful discussions on the latest in biotech and data science.Subscribe and leave a review if you enjoyed this episode! Sponsored by… This episode is brought to you by CorrDyn, the leader in data-driven solutions for biotech and healthcare. Discover how CorrDyn is helping organizations turn data into breakthroughs at CorrDyn.

Officers from a UK medicine regulator raided two sites in February during an ongoing investigation into a criminal network that manufactures and distributes unlicensed weight-loss medicines, also known as “skinny jabs.”The Medicines and Healthcare products Regulatory Agency (MHRA) stated that the operation, which targeted farm and residential properties in Lincolnshire and Nottinghamshire, resulted in the seizure of nearly 2,000 doses of unauthorized weight-loss medicines, including retatrutide, tirzepatide and peptide products. The agency added that officers also confiscated manufacturing equipment, suspected pharmaceutical ingredients, packaging and commercial vehicles. #MHRA #WeightLossDrugs #SkinnyJabs #IllegalMedicines #DrugSafety #PublicHealth #Tirzepatide #Retatrutide #GLP1 #PharmaceuticalCrime #HealthcareNews #MedicalRegulation #CounterfeitDrugs #FDA #UKNews #DrugEnforcement #HealthRisks #RegulatoryAction #LawEnforcement #MedicineSafety

In this conversation we hear about Kola's journey as self-taught coder, business school, learning by doing, and how he is self-funding one person AI company for doctors: Kola Tytler's parallel journey as an NHS Doctor while building pioneering and potentially world changing business is inspiring. Listen in on a remarkable conversation between host Richard Lucas and Kola Tytler, now a qualified doctor who taught himself to code. We explore the roots of his entrepreneurial activity, despite knowing he wanted to be a doctor from a young age. the influence and opportunities of being an immigrant from a different background as he went to medical school in London. his first venture selling event tickets via a Facebook platform, scaling a fashion blog with millions of followers, and launching and exiting the successful Dropout retail business in Milan Lessons of having investors who were not always aligned How he dealt with realising that he might have a bigger financial opportunity through dropping out of his studies. The benefits and limitations of bootstrapping when you have the resources to put together a great team The impact of both his formal business school education and self tuition via online resources like Y Combinator, and prominent SV figures like Steve Jobs and Bill Gates. The ambition and vision for his self funded AI platform for doctors iatroX which provides clinical guidance to over 20,000 users. Kola's journey is a masterclass in calculated risk and relentless drive, Kola shares the critical lessons he has learned from his triumphs and challenges. Through insightful questions, Richard draws out the key takeaways on finding balance, the importance of a strong team, understanding domain expertise, and the necessity of continuous business education. This episode is packed with inspiration for anyone looking to bridge diverse passions and build a high-impact venture. About Kola Dr Kola Tytler – Doctor/MBA & full-stack developer MBBS @ King's College London Certificates in Law & Business (LSE & Imperial) MBA (with merit) @ University of Birmingham MSt Entrepreneurship @ University of Cambridge ‘26 Forbes 30 under 30 Europe, Forbes 100 under 30 Italy IBM-certified AI Engineer & MENSA member Founder of YEEZY Mafia, dropout, & HypeAnalyzer Links iatroX is a UKCA-marked, MHRA-registered medical device. It acts as an AI‑driven assistant that centralizes clinical guidelines offering: 1 quick Q&A, 2 structured brainstorming, and 3 an adaptive quiz engine for medical students. Kola Tytler's Linkedin Kola Tytler's personal websiteDrop Out MilanoHype Analyzer CAMentrepreneurs Learn more about your ad choices. Visit megaphone.fm/adchoices Support our show by becoming a premium member! https://newbooksnetwork.supportingcast.fm/new-books-network

In this conversation we hear about Kola's journey as self-taught coder, business school, learning by doing, and how he is self-funding one person AI company for doctors: Kola Tytler's parallel journey as an NHS Doctor while building pioneering and potentially world changing business is inspiring. Listen in on a remarkable conversation between host Richard Lucas and Kola Tytler, now a qualified doctor who taught himself to code. We explore the roots of his entrepreneurial activity, despite knowing he wanted to be a doctor from a young age. the influence and opportunities of being an immigrant from a different background as he went to medical school in London. his first venture selling event tickets via a Facebook platform, scaling a fashion blog with millions of followers, and launching and exiting the successful Dropout retail business in Milan Lessons of having investors who were not always aligned How he dealt with realising that he might have a bigger financial opportunity through dropping out of his studies. The benefits and limitations of bootstrapping when you have the resources to put together a great team The impact of both his formal business school education and self tuition via online resources like Y Combinator, and prominent SV figures like Steve Jobs and Bill Gates. The ambition and vision for his self funded AI platform for doctors iatroX which provides clinical guidance to over 20,000 users. Kola's journey is a masterclass in calculated risk and relentless drive, Kola shares the critical lessons he has learned from his triumphs and challenges. Through insightful questions, Richard draws out the key takeaways on finding balance, the importance of a strong team, understanding domain expertise, and the necessity of continuous business education. This episode is packed with inspiration for anyone looking to bridge diverse passions and build a high-impact venture. About Kola Dr Kola Tytler – Doctor/MBA & full-stack developer MBBS @ King's College London Certificates in Law & Business (LSE & Imperial) MBA (with merit) @ University of Birmingham MSt Entrepreneurship @ University of Cambridge ‘26 Forbes 30 under 30 Europe, Forbes 100 under 30 Italy IBM-certified AI Engineer & MENSA member Founder of YEEZY Mafia, dropout, & HypeAnalyzer Links iatroX is a UKCA-marked, MHRA-registered medical device. It acts as an AI‑driven assistant that centralizes clinical guidelines offering: 1 quick Q&A, 2 structured brainstorming, and 3 an adaptive quiz engine for medical students. Kola Tytler's Linkedin Kola Tytler's personal websiteDrop Out MilanoHype Analyzer CAMentrepreneurs Learn more about your ad choices. Visit megaphone.fm/adchoices Support our show by becoming a premium member! https://newbooksnetwork.supportingcast.fm/medicine

Today, the MHRA puts the brakes on the UK's PATHWAYS puberty blocker trial work while safety concerns get addressed, the UK's space-weather mission heads toward its launch site (because satellites don't protect themselves), and NASA's Artemis II rocket gets rolled back for more fixes — yes, really. After that: a quick cyber patch warning, a punchy Arc Raiders update, and Samsung's Unpacked week landing like a new phone season jump-scare. More on standard.co.uk Hosted on Acast. See acast.com/privacy for more information.

By 2028, SGS CDMO Solutions' MHRA and FDA-inspected site in Wales will operate entirely without paper. For biotech and Pharmaceutical sponsors, this provides a better way to run development and manufacturing projects.“If it can't be done digitally, then we don't want to do it,” says Paul Broomhead, Head of Site. The result is a digitally native CDMO where data, processes, and decisions live in one.Paul Broomhead, Head of Site at SGS CDMO Solutions in Wales, is leading an ambitious digital transformation. In this exclusive interview, Paul reveals why SGS is making a commitment to digital infrastructure, how the transformation creates competitive advantages for biotech sponsors, and what “digitally native” manufacturing actually means in practice for pharmaceutical development and commercialization.Read more

File on 4 Investigates whether the risks associated with the popular hair loss drug Finasteride are understood, after a 2024 review by UK drug safety regulator the MHRA prompted by a lack of awareness of the drug's side-effects. Finasteride's most common side effects are reduced libido and erectile dysfunction, affecting more than one in a hundred patients. Some people also report low mood and suicidal thoughts. As Johnny I'Anson discovers some hair loss clinics are failing to adequately warn customers at the consultation stage of the sexual side effects assocated with Finasteride.The drug, which is highly effective at halting hair loss, is only available by private prescription for hair loss purposes. But File on 4 Investigates discovers influencer accounts on the social media site TikTok promoting finasteride, also offering discounts for Finasteride with Manual, a popular UK men's health company. A lawyer expert told us that the videos we found constituted advertising. As a prescription-only medicine, it's illegal to advertise finasteride to the public like this. This programme flagged the videos to TikTok, and they have since been removed. The company has also banned three of the accounts promoting the drug, saying they breached its rules. Manual said it was not involved in the making of the influencers' videos.Presenter: Johnny I'Anson Producer: Rob Byrne Technical Producer: Richard Hannaford Production Co-ordinator: Tim Fernley Editor: Tara McDermottDetails of organisations offering help and support with mental health are available at bbc.co.uk/actionline

In the second hour of Race Central with Kurt Hansen, "Photo" Joe Starr joins ther show to talk the latest new in MHRA and INdy car racing. With rumors of European models joing Indy car racing. Also Kurt discusses speculation of Bandimere Speedway ownership siging with IHRA which would shake up the racing world with their former association with NHRA.

These have been the biggest scams we've seen this year - plus our best advice on how to avoid them. In this episode of Which? Shorts, we countdown the the scams we've come across most often in 2025, explain what you can do to keep yourself safe online, and how to react if you've been targeted by a fraudster. Read more about the biggest scams of 2025 & sign up for our free Scam Alerts service Become a Which? member for 50% off the usual price Click here to report a scam to Action Fraud Search for a number on who-called & look up a website on who.is Check whether the product or medicine is approved on the MHRA website

OS Therapies Inc (NYSE-A:OSTX) chief business officer Gerald Commissiong talked with Proactive's Stephen Gunnion about the company's recent regulatory progress for its OST-HER2 immunotherapy platform targeting osteosarcoma. Commissiong detailed how OS Therapies acquired rights to a listeria-based therapy originally developed by Advaxis and how this technology has evolved into a precision platform targeting HER2-expressing cancers. The OST-HER2 approach uses attenuated listeria engineered to infect cancer cells, triggering a robust immune response. “Basically, once infected with the listeria, our vector releases additional cancer antigens,” Commissiong explained, noting how this mechanism amplifies immune activity via T cells, NK cells, and dendritic cells. He outlined progress with regulators, including a Type C meeting with the FDA, pre-marketing discussions with the MHRA in the UK, and a meeting with the European CHMP. “The tenor of those conversations… is around trying to find a pathway to get this drug into patients' hands sooner,” he said, adding that discussions included using historical control data and biomarkers as surrogate endpoints for accelerated approval. Commissiong also revealed the broader pipeline, including constructs targeting HPV-driven cancers and prostate cancer. Key next steps include biomarker analysis and initiation of a confirmatory study. Visit Proactive's YouTube channel for more biotech and pharma interviews. Don't forget to like this video, subscribe to the channel, and enable notifications for future content. #OSTHER2 #OSTherapies #Osteosarcoma #CancerImmunotherapy #FDAapproval #Biotech #ListeriaTherapy #CancerResearch #AcceleratedApproval #OncologyInnovation #HPVcancer #ProstateCancer #MHRA #CHMP

Richie is joined by Hedley Rees. Hedley is a pharmaceutical supply-chain specialist with decades of experience in the industry. He's held senior roles at companies including Bayer and Johnson & Johnson. These days he runs PharmaFlow, a consultancy firm advising on drug manufacturing and logistics. Back in 2020 he raised serious concerns about the COVID-19 vaccine supply chain and the total absence of regulatory oversight during the rollout of the jabs. Hedley knew the jabs would cause widespread harm. Despite a concerted campaign of censorship, Hedley never gave up. Since 2024, he has been writing to the UK regulator - MHRA - and demanding answers. Follow and support Hedley here:https://substack.com/@hedleyrees1https://www.facebook.com/hedley.rees.967

Medicus Pharma CEO Dr Raza Bokhari joined Steve Darling from Proactive to announce that the company has received full regulatory and ethical approvals in the United Kingdom to expand its ongoing Phase 2 clinical trial evaluating its Doxorubicin Microneedle Array technology for the non-invasive treatment of basal cell carcinoma of the skin. The study, known as SKNJCT-003, is already underway across nine clinical sites in the United States and will now broaden to include additional sites in the U.K., following this latest approval milestone. Dr. Bokhari explained that approvals were issued by the Medicines and Healthcare products Regulatory Agency , the Health Research Authority (HRA), and the Wales Research Ethics Committee. The MHRA's authorization came after a comprehensive scientific review of the Investigational Medicinal Product Dossier and clinical protocol, while WREC issued a favorable ethical opinion. The HRA subsequently granted study-wide governance approval, confirming full compliance with U.K. Good Clinical Practice (GCP) and National Health Service (NHS) capacity and capability standards. The Phase 2 trial is designed as a randomized, double-blind, placebo-controlled, multi-center study enrolling up to 90 patients with BCC. It aims to evaluate the efficacy and safety of two dose levels of D-MNA compared to a placebo control. Participants will be randomized into three groups in a 1:1:1 ratio, a placebo-controlled group, a low-dose group, and a high-dose group. Dr. Bokhari highlighted that the 200 μg high-dose level represents the maximum dose previously tested in Medicus Pharma's Phase 1 safety and tolerability study, which was successfully completed in March 2021. The company views the U.K. expansion as a significant step toward advancing its novel microneedle drug delivery platform and potentially transforming the treatment landscape for non-melanoma skin cancers through a minimally invasive, localized therapy that may reduce the need for surgery and improve patient outcomes. #proactiveinvestors #nasdaq #mdcx #tsxv #mdcx #pharma #Biotech #CancerTreatment #ClinicalTrials #FDAApproval #SkinCancer #HealthcareInnovation #Investing #MedicalResearch #SkinCancer #BasalCellCarcinoma #BiotechNews #CancerResearch #GorlinSyndrome #BasalCellCarcinoma #CompassionateUse #FDAApproval #RareDiseaseTreatment #NoninvasiveTherapy #BiotechNews

We're joined by representatives from the MHRA to discuss how NHS patients will be able to access medicines up to 6 months faster through an aligned MHRA and NICE pathway. Watch the webinar here: https://www.youtube.com/watch?v=FVBVYTUc2Q4

Send us a textIn this powerful episode, I sit down with Holly, a remarkable professional who transforms her harrowing journey through anorexia into a mission of mental health advocacy. From battling a life-threatening eating disorder as a teenager to now leading digital mental health technology regulation, Holly shares an intimate and inspiring story of survival, resilience, and purpose. Holly Coole is Senior Manager for Digital Mental Health at the MHRA and lead forthe Wellcome-funded project to explore the clinical evaluation and regulation ofdigital mental health technologies. She has also worked for NHS Supply Chain asthe Patient Safety and Innovation Manager. Holly has a background in psychiatricnursing, previously working for Nottinghamshire Healthcare NHS Foundation Trustas a Community Psychiatric Nurse for several years along with experience in anumber of mental healthcare services such as inpatient forensic, older adults andchild and adolescent mental health. Holly has also undertaken training in cognitivebehavioural therapy and brings her own insights to lived experience of mental health.Holly sincerely discusses her struggles with perfectionism, her cyclical recovery, and how she's turned her most challenging experiences into a force for positive change.This episode offers a raw, honest look at mental health, breaking stigmas, and finding hope when all seems lost. You will be moved by Holly's courage and insights into self-compassion, personal growth, and the importance of supporting those battling mental health challenges.If you've ever felt trapped by your own expectations, this episode will set you free.Watch it on YouTube: https://youtu.be/6MonJ7Rnca4Don't forget to "Like and Subscribe", so we can reach more people to help.Visit www.mindandmood.co.uk, email info@mindandmood.co.ukor call us on +44 (0)207 183 6364 to find out more.#MentalHealthAwareness #EatingDisorderRecovery #SelfCompassion #MentalHealthJourney #WellnessTechnology #Resilience #BreakTheStigma #MentalHealthAdvocacy #PersonalGrowth #SelfLove #RecoveryStory #MentalHealthTech #Perfectionism #Healing #WomensHealth #MentalWellness #SurvivorsStory #DigitalHealth #MindBodyHealing #InspirationalStorySupport the show

There's been a rare IPO filing on NASDAQ as LB Pharma looks to test the market during a year that has seen little activity among U.S. biotechs even as green shoots continue to appear on the Hong Kong stock exchange. On the latest BioCentury This Week podcast, BioCentury's analysts discuss the market for biotech IPOs on NASDAQ and in Hong Kong.The analysts then assess FDA's about-face on Stealth BioTherapeutics' Barth syndrome therapy, putting the decision in the context of a changing regulatory agency; and a BioCentury Guest Commentary that argues that the university-industry engine that drives U.S. innovation is under attack. Also mentioned on this week's podcast: BioCentury's 33rd Back to School package, which reimagines FDA; the upcoming 12th China Healthcare Summit in Shanghai; the evolution of dealmaking in China; and Annalisa Jenkins' take on MHRA and the U.K. biotech ecosystem on The BioCentury Show.View full story: https://www.biocentury.com/article/656849#Biotech #IPO #Pharma #FDA #RareDisease #Biopharma #DrugDevelopment #HealthcareInnovation #HongKongIPO00:00 - Introduction 02:48 – LB Pharma Tests IPO Market07:01 – Hong Kong IPO Momentum09:53 – China Summit Preview13:40 – FDA Reversal on Stealth Bio18:15 – Bayh-Dole Clash & Innovation ThreatsTo submit a question to BioCentury's editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

Microsoft's head of artificial intelligence said he's being “kept awake at night” as more people report suffering from “AI psychosis”.David Shrier, Professor of Practice, AI & Innovation at Imperial College London, told us "if someone is experiencing a psychotic episode, the AI might inadvertently reinforce the psychosis."And, scientists have found our internal compass.Russell Epstein, Professor of Psychology at the University of Pennsylvania, told us how they used neuroimaging and virtual reality to identify two brain regions that help humans maintain their sense of direction while moving around.Also in this episode:-We speak to Lynda Scammell, head of borderline at the MHRA, about the product warning relating to Nutrition Ignition Magnesium Glycinate Gummies-US gamers will see a price hike for Sony's PlayStation 5 console-Google have unveiled their 10th generation of phone.-A seabird that only poops while flying Hosted on Acast. See acast.com/privacy for more information.

Becoming an ISO consultant isn't a career path many aspire to, rather it's one often stumbled on after being tasked with either implementing or maintaining a Standard for a business. We're continuing with our latest mini-series where we introduce members of our team, to explore how they fell into the world of ISO and discuss the common challenges they face while helping clients achieve ISO certification.   In this episode we introduce Minoo Agarwal, a QHSE Consultant at Blackmores, to learn about her journey of following in her father's footsteps towards ISO Standards Management, and what drives her to help clients on their ISO journey.   You'll learn ·      What is Minoo's role at Blackmores? ·      What does Minoo enjoy outside of consultancy? ·      What path did Minoo take to become an ISO Consultant? ·      What is the biggest challenge she's faced when implementing ISO Standards? ·      What is Minoo's biggest achievement?   Resources ·      Isologyhub   In this episode, we talk about: [02:05] Episode Summary – We introduce Minoo Agarwal, a QHSE Consultant here at Blackmores, to discuss her journey towards becoming an ISO consultant who specialises in ISO 14001, ISO 9001, ISO 45001 and ISO 27001.    [03:50] What is Minoo's role at Blackmores? Minno's official job title is QHSE Consultant. She is the ISO14001:2015 standard champion and a Mental Health First Aider for Blackmores. Ultimately, Minoo supports clients with embedding Management System into the heart of their companies. Her work with them typically consists of: ·      Conducting internal audits ·      Management review ·      Consultancy days or document review days Essentially doing whatever it takes to getting the management system to be at a suitable level to pass external audits.   [05:05] What does Minoo enjoy doing outside of consultancy?: Minoo's free time is mostly taken up by her dear son, Aarav. He's very young at the moment; and so Minoo makes sure that any of her input into his life is to ensure that he is successful in whatever career he chooses. In addition, Aarav has a very busy social life! So, she's makes sure her gets plenty of time to play with his friends. Minoo is also a bit of a foodie, enjoying eating out when possible. She also enjoys reading books by authors such as Jay Shetty and the Sad Guru. [07:10] What was Minoo's path towards becoming an ISO Consultant?: Minoo, like many of our consultants, didn't know that she would become a consultant. The opportunity was presented by Mel Blackmore via LinkedIn in 2019, on April 1st of all dates! Minoo's passion for this field arose from her father, Mr Hardial Agarwal, who is very well known in the industry. He worked as a consultant for Crayola for many years, travelling abroad to meet with various suppliers and international branches of the company. His work always held an air of mystery to Minoo as a child, and she become more curious about his role in later years, even attending CQI meetings with her father to learn more. She started her career in 2006 as a Quality, Environmental, Health & Safety Administrator and since then really never looked back, progressing from role to role from roles like Quality Associate, to a Business Quality Control Officer, to a QESH Auditor, to HSQE Compliance Manager and then as a Head of Quality.  Each role gave her a different experience of life. As a result, she has worked in many industries ranging from electronics to logistics to pharma and even automotive and IT. She feels very fortunate to gain experience and knowledge from a range of industries from her previous roles, and now more so from Blackmores where this knowledge develops further. [12:35] What is Minoo's favourite aspect of being a Consultant? – Minoo genuinely loves her role as a consultant at Blackmores, it was hard to narrow down a specific aspect. That being said, Minoo loves to hear when a client of hers has passed their surveillance and re-certification audits, especially if it was with no findings. It's a return on their combined effort as a team to get recognition of that fact from a certification body. She also enjoys the teamwork involved, often being seen as a real member of the client's team. As a consultant, a bid part of your role is building strong working relationships, which makes the whole process run a lot more smoothly. She also takes a bit of joy in being able to be a bit bossy, though all the guidance is with the best intentions. [15:40] What Standards does Minoo specilaise in and why? Starting with: ·      ISO 9001 Quality Management: A core foundation that many businesses start with when diving into the world of ISO Standards. This is an essential one for any ISO consultant and is often the first Implementation experience for many who go on to become ISO consultants. ·      ISO 14001 Environmental Management: Minoo is our Standards champion for this Standard. This involves keeping on-top of any changes to the Standard and creating internal training material for the team.    ·      ISO 45001 Health and Safety Management: A few of Minoo's previous roles involved health and safety compliance, so she learned a lot about ISO 45001 and general risk management along the way.    ·      ISO 27001 Information Security Management: This one was a necessity to learn when joining Blackmores. Many of our clients have integrated management systems with multiple ISO certifications, most of which include ISO 27001. Minoo also has some experience with MHRA regulations and TS 16949. [18:20] What is the biggest challenge Minoo had faced during a project and how did she overcome it?: Minoo stresses that it's fundamental to fully understand the requirements of the client from the very onset of the project, if that is understood then there will be no challenges during a project. She provides two incidents in particular that stood out: Scope creep: There was an incident when there was a misunderstanding of the work agreed, and the client had anticipated much more than what was agreed. As a result, Minoo had to complete the work in her own time to meet their expectations. Personal bias: There have been incidents not related to projects but external audits when external assessors start to audit the client by providing what they believe their interpretations is of the standard is the only way that it can be implemented. Forgetting the fact that Standards are built to be flexible in the way they are implemented. They can get very fussy about the exact way documentation should be laid out and worded, which is obviously not a specific stipulation within a Standard. Minoo has overcome this by confronting the assessor at the time and asking them to explain where in the standard it say that – basically keeping them in check. [21:30] What is Minoo's proudest achievement?  On the work side, Minoo is always proud to hear when a client has passed their Surveillance and Recertification Audits with no or few findings. On the personal side, it's her son who she lovingly dubs as her masterpiece. She's already instilling the values of health and safety into him, and he's always looking out for others. She couldn't be prouder to watch him grow up into his own person. If you'd like any assistance with implementing ISO standards, get in touch with us, we'd be happy to help! We'd love to hear your views and comments about the ISO Show, here's how: ●     Share the ISO Show on Twitter or Linkedin ●     Leave an honest review on iTunes or Soundcloud. Your ratings and reviews really help and we read each one. Subscribe to keep up-to-date with our latest episodes: Stitcher | Spotify | YouTube |iTunes | Soundcloud | Mailing List

Send us a textThe Most Overlooked Risk in AI for Pathology? It's Not What You Think…Welcome, my trailblazing digital pathologists! In this episode, I dive headfirst into the regulatory maze of Artificial Intelligence (AI) in pathology, covering global frameworks, safety risks, ethics, and the future of software as a medical device. While regulation might not be the flashiest part of AI, ignoring it could cost us innovation—or worse, patient safety.We're on Part 5 of our 7-part AI in Pathology series, and this one's vital for anyone developing, using, or simply curious about AI and machine learning tools in healthcare.If you thought regulation was boring, think again—it's what separates a helpful algorithm from a dangerous black box.

Send us a textGrab your trainers, your dog lead, or your drink of choice-with ice-  and join us for some free CPD as we have another relaxed round up of recent Red Whale primary care Pearls of wisdom.  In the first of two episodes this month, Fi and Nik discuss: Contraception, weight and GLP-1s: “Women on ‘skinny jabs' must use effective contraception, MHRA urges in latest guidance” – Government press release, 5 June 2025. We share our newly-updated article on contraception and weight, which includes this MHRA guidance.There is NO safe level of drinking: the missed message? Since 2016, the message from the UK Chief Medical Officer is clear: there is no safe level of drinking. This was a big change from previous messaging that has perhaps not fully reached the professional or public consciousness.     Listen as soon as you can to ensure you have full access to all the free resources. More Pearls from July will be covered next week.Alcohol    The National Institute on Alcohol Abuse and Alcoholism - youth screening tool (for children aged 9–18y)  E-learning for healthcare - alcohol identification and brief advice   Send us your feedback podcast@redwhale.co.uk or send a voice message Sign up to receive Pearls here. Pearls are available for 3 months from publish date. After this, you can get access them plus 100s more articles when you buy a one-day online course from Red Whale OR sign up to Red Whale Unlimited. Find out more here. Follow us: X, Facebook, Instagram, LinkedInDisclaimer: We make every effort to ensure the information in this podcast is accurate and correct at the date of publication, but it is of necessity of a brief and general nature, and this should not replace your own good clinical judgement, or be regarded as a substitute for taking professional advice in appropriate circumstances. In particular, check drug doses, side-effects and interactions with the British National Formulary. Save insofar as any such liability cannot be excluded at law, we do not accept any liability for loss of any type caused by reliance on the information in this podcast....

George and Louise break down the hot topics of the day, including:AI Scribes now regulated as medical devices in the UK - all pilots must cease until MHRA approval UK Government announces £10 billion digital health investment and AI Early Warning System NHS 10-Year "Fit to the Future" Plan - three major shifts including analogue to digital transformation Cybersecurity failures at WA's PathWest and UK's Synnovis directly impacting patient safety?Positive AI developments: FDA approves breast cancer risk prediction tool, Australian chemotherapy dosing AIHow does Australia go about integrating 300,000+ allied health professionals into the national digital health strategy? That topic and more are covered in the chat with our guest Anita Hobson-Powell, the inaugural Chief Allied Health Officer at the Australian Government Department of Health, Disability and Ageing.Connect with Anita on LinkedInResources:NHS 10 year health plan for England. Fit to the Future LinkNational Allied Health Digital Uplift. Responses due 20 July LinkHealth Connect Australia Strategy, Architecture and Roadmap to enable health information exchange LinkANU Study on the Future of Digital Health EOI LinkVisit Pulse+IT.news to subscribe to breaking digital news, weekly newsletters and a rich treasure trove of archival material. People in the know, get their news from Pulse+IT – Your leading voice in digital health news.Follow us on LinkedIn Louise | George | Pulse+ITFollow us on BlueSky Louise | George | Pulse+ITSend us your questions pulsepod@pulseit.newsProduction by Octopod Productions | Ivan Juric

The government's health agency, the MHRA, is warning women that they shouldn't use weight loss jabs while pregnant and that the drugs can also affect the reliability of the pill, which has led to a rise in so-called "Ozempic babies".   On today's Sky News Daily Niall Paterson talks to Dr Nikita Kanani, a GP and former medical director for primary care at NHS England, about the risks, whether there are other concerns about using them and if there should be tighter restrictions on online sales of the jabs.  Producer: Emily Hulme Editor: Wendy Parker 

FREEDOM - HEALTH - HAPPINESSThis podcast is highly addictive and seriously good for your health.SUPPORT DOC MALIK To make sure you don't miss any episodes, have access to bonus content, back catalogue, and monthly Live Streams, please subscribe to either:The paid Spotify subscription here: https://creators.spotify.com/pod/show/docmalik/subscribe The paid Substack subscription here: https://docmalik.substack.com/subscribeThank you to all the new subscribers for your lovely messages and reviews! And a big thanks to my existing subscribers for sticking with me and supporting the show! ABOUT THIS CONVERSATION: In this episode, I speak with Cheryl, who brings insider experience from the pharmaceutical world and a deeply analytical mind to everything that's unfolded over the past few years.We dive into the role of the MHRA, the UK's drug regulator, and explore whether it's truly serving public health or merely rubber-stamping products handed to it by pharmaceutical companies. Cheryl shares her concerns about the lack of transparency, the failures of pharmacovigilance, and how freedom of information requests have revealed troubling gaps in regulatory oversight, especially in relation to the COVID-19 vaccine rollout.We talk about mRNA technology, gene therapy, and the disturbing trend of pushing products to market without adequate long-term safety data. Cheryl also highlights the yellow card system, supposedly in place to track adverse effects, which is shockingly underused and almost unknown to most doctors.See my substack for more information.I hope you enjoy this episode.Much love, as always.Doc MalikLinksWebsite https://substack.com/@cherylgrainger IMPORTANT INFORMATIONCONSULTATION SERVICEIn a world of rushed 7-minute consultations and endless referrals, I offer you something rare: time, context, and clear guidance.As your health advocate, I can help you:Understand your diagnosis and decode medical jargonDecide who to see: GP, specialist, osteopath, physio, accupuntcurist, homeopath etc?Break down treatment plans in plain, easy to understand non jargon EnglishPrepare for surgery, understand your risks, obtain true informed consent, and optimise yourself pre-op Recover from surgery, advise you how to heal faster and quicker and minimise post-op complicationsManage chronic illness with lifestyle, mindset, and dietary changesExplore holistic options that complement conventional careImplement lifestyle changes like fasting, stress reduction, or movementAsk better questions, and get real answersGet an unbiased second opinionReady to Take Control?If you're navigating a health concern, preparing for a big decision, or simply want to feel more confident in your path forward, I'd love to support you.Book here https://docmalik.com/consultations/ Because it's your body, your life, and your future. Let's make sure you're informed and heard.WaterpureI distill all my water for drinking, washing fruit and vegetables, and cooking. If you knew what was in tap water, so would you!https://waterpure.co.uk/docmalik BUY HERE TODAYHunter & Gather FoodsSeed oils are inflammatory, toxic and nasty; eliminate them from your diet immediately. Check out the products from this great companyhttps://hunterandgatherfoods.com/?ref=DOCHG BUY HERE TODAYUse DOCHG to get 10% OFF your purchase with Hunter & Gather Foods.IMPORTANT NOTICEIf you value my podcasts, please support the show so that I can continue to speak up by choosing one or both of the following options - Buy me a coffee If you want to make a one-off donation.Doc Malik Merch Store Check out my amazing freedom merch

On this week's episode, Lisa Moneymaker (SVP, Head of Strategic Customer Engagement, Medidata Solutions) and Adam Aten (Legislative & Regulatory Policy Lead, Verily) join the podcast to discuss how the clinical research industry must use insights from the past to better prepare our AI models and other technologies to meet the needs of patients in the present and future. They dive deeper into the role that collaboration between technologists and clinical scientists can play in helping to reduce bias in our AI models, what legislators and regulators should be keeping top of mind as they write new rules of the road for AI and ML, and ACRO's ongoing efforts to promote the responsible use of AI in clinical research.

Welcome to Health Check, the podcast that takes the temperature on regulatory developments affecting the life sciences and healthcare sector in the UK, and offers a prescription to these challenges. In this episode, Osborne Clarke's regulatory lawyers Anna Lundy and Peter Rudd-Clarke, explore regulatory reform in the AI and healthcare world, compare events in the UK with the EU approach, and ask how has the new UK government and MHRA are supporting innovation.

In this final episode of our mini-series on the AstraZeneca Covid vaccine, Investigations Editor Claire Newell explores whether the MHRA, the regulatory agency for drugs, has protected patients. She hears from families about the long-term consequences of a rare adverse reaction to the jab, and whether they have received enough support from the Government.Written by: Claire NewellProducer: Jack BoswellExecutive Producer: Adélie Pojzman-Pontay Hosted on Acast. See acast.com/privacy for more information.

Rare condition research is evolving, and patient communities are driving the breakthrough. In this special Rare Disease Day episode, we explore the challenges and opportunities shaping the future of rare condition therapies. From groundbreaking gene therapy trials to the power of patient-driven research, our guests discuss how collaboration between families, clinicians, researchers, and regulators is paving the way for faster diagnoses, equitable access to treatments, and innovative approaches like nucleic acid therapies and CRISPR gene editing. With insights from Myotubular Trust, we follow the journey of family-led patient communities and their impact on advancing gene therapy for myotubular myopathy - showcasing how lived experience is shaping the future of medicine. However, while patient-driven initiatives have led to incredible progress, not every family has the time, resources, or networks to lead these research efforts. Our guests discuss initiatives like the UK Platform for Nucleic Acid Therapies (UPNAT), which aims to streamline the development of innovative treatments and ensure equitable access for everyone impacted by rare conditions. Our host Dr Ana Lisa Tavares, Clinical lead for rare disease at Genomics England, is joined by Meriel McEntagart, Clinical lead for rare disease technologies at Genomics England, Anne Lennox, Founder and CEO of Myotubular Trust and Dr Carlo Rinaldi, Professor of Molecular and Translational Neuroscience at University of Oxford. "My dream is in 5 to 10 years time, an individual with a rare disease is identified in the clinic, perhaps even before symptoms have manifested. And at that exact time, the day of the diagnosis becomes also a day of hope, in a way, where immediately the researcher that sent the genetics lab flags that specific variant or specific mutations. We know exactly which is the best genetic therapy to go after." You can download the transcript, or read it below. Ana Lisa: Welcome to Behind the Genes.    [Music plays]  Anne: What we've understood is that the knowledge and experience of families and patients is even more vital than we've all been going on about for a long time. Because the issue of there being a liver complication in myotubular myopathy has been hiding in plain sight all this time, because if you asked any family, they would tell you, “Yes, my son has had the odd liver result.”  There were some very serious liver complications but everybody thought that was a minor issue, but if we are able to engage the people who live with the disease and the people who observe the disease at a much more fundamental level we may be able to see more about what these rare genes are doing.  [Music plays]  Ana Lisa: My name is Ana Lisa Tavares, I'm Clinical Lead for Rare Disease research at Genomics England and your host for this episode of Behind the Genes. Today I'm joined by Anne Lennox, Founder and CEO of the Myotubular Trust, Dr Meriel McEntagart, an NHS consultant and Clinical Lead for Rare Disease Technologies at Genomics England, and Dr Carlo Rinaldi, Professor of Molecular and Translational Neuroscience at the University of Oxford.    Today we'll be hearing about the importance of involving the patient community, particularly as new rare therapies are developed, and discussing the forward-facing work that's happening that could have potential to unlock novel treatments for many rare conditions.  If you enjoy today's episode we'd love your support. Please like, share and rate us on wherever you listen to your podcasts. Thank you so much for joining me today.  Please could you introduce yourselves.   Anne: I'm Anne Lennox, I'm one of the founders of the Myotubular Trust, a charity that raises research funds for and supports families affected by the rare genetic neuromuscular disorder myotubular myopathy.  Meriel: I'm Meriel McEntagart, I'm a consultant in clinical genetics in the NHS and I have a special interest in neurogenic and neuromuscular conditions.  Carlo: Hi, I'm Carlo Rinaldi, I'm Professor of Molecular and Translational Neuroscience at the University of Oxford. I'm a clinician scientist juggling my time between the clinic and the lab where we try to understand mechanisms of diseases to develop treatments for these conditions.  And I'm also here as a representative of the UK Platform for Nucleic Acid Therapies, UPNAT. Thanks for your invitation, I'm very pleased to be here.  Ana Lisa: Thank you. Meriel, I'd love you to tell us a bit about your work and how you met Anne, how did this story start?  Meriel: Thank you. Well prior to being a consultant in clinical genetics, I spent 2 years as a clinical research fellow in neuromuscular conditions, and as part of that training I worked on a project where the gene for myotubular myopathy had just been identified, and so there was a big international effort to try and come up with sort of a registry of all the genetic variants that had been found as well as all the clinical symptoms that the affected patients had, and then do kind of a correlation of the particular variant mutation with symptoms.   I worked when I was training to be a clinical geneticist because of my interest in neuromuscular conditions so when I eventually became a consultant at St George's Hospital I was actually interviewed by the Professor of Paediatrics and he knew Anne and her son, when Anne was looking for more information about the condition he suggested that perhaps I might be a good person for Anne to talk to.  Ana Lisa: Thank you. Interesting connections. Anne, can you tell us your story and how this led you to found the Myotubular Trust?  Anne: Yes, thanks Ana-Lisa.  Well, as many families will tell you when they're newly diagnosed with a rare disease, you go from knowing nothing about a condition to being one of the few deep experts in that condition because there are so few deep experts. So this happened to us in 2003 when our son, Tom, was born, and when he was born he was floppy and his Apgar scores, the scores they do on new-born babies, were pretty poor, and before long we knew that it was more than just momentary issues at birth.  And, cutting a very long story short, 5 weeks later he was diagnosed with this very rare neuromuscular genetic disorder that we didn't know we had in the family.  We were told that this was a very serious diagnosis.    At that time – more than 20 years ago – over 80% of those boys didn't make it to their first birthday and the stark statistic we had in our head a lot was that only 1% made it past the age of 10. And that has changed due to better ventilator and breathing equipment, etc, but at the time we expected that he might not make it to his first birthday.    We were very lucky, we had Tom longer than one year, we had him for nearly 4 years, 4 very lovely years where it was tough, but he was a really lovely member of our family.  Despite being really weak he managed to be incredibly cheeky and bossy, and he was a great little brother for his big sister. We were also very lucky that he was being looked after by Professor Francesco Muntoni, who is Head of the Paediatric Neuromuscular Service at Great Ormond Street. And, like Carlo, he is a clinical researcher and actually that I found to be amazing as a family member because you knew what was happening out there and Professor Muntoni, other than living with the reality day to day you want to know where things are going.    We began to realise that back then 20 years ago the more common rare neuromuscular diseases were finally beginning to get some fundamental research funds, like Duchenne, spinal muscular atrophy, and Professor Muntoni was very good at explaining to lay non-scientific parents like us that one day the technologies that would lead to a cure, that would re-engage proteins for other conditions and would translate down eventually into the possibility of replacing myotubularin, which is the protein not being produced or not being produced enough in myotubular myopathy. And then we began to understand actually what the barriers to that would be, that translating developments in more common, or let's say more prevalent conditions, would be hard to do without some translation research being done; you could not just not lag years behind, you could lag decades behind if you haven't done some other work.    So, I met Wendy Hughes, another mother, of a boy called Zak who was a few years older than Tom, and these were the days before social media, and it was amazing to be in contact with another family going through something similar and we had great conversations. But then they were also looked after by Professor Muntoni and we particularly began to develop the idea as 2 families that we might be able to raise some research funds towards this concept of keeping pace with the scientific developments.  And then we discovered there was no charity we could channel those funds through. Even the umbrella body for neuromuscular diseases who were covering 30 to 40 conditions, frankly, they just couldn't trickle their funding down into investing in every neuromuscular disease, and slowly but surely it dawned on us that if we did want to make that difference we were going to have to set up our own charity.   So that's what we eventually did and back in 2006, we founded what was actually the first charity in Europe dedicated to myotubular myopathy – luckily, more have come along since – and we were dedicated to raising research funding. In fact, it wasn't our goal to set up another charity but around that time, about a year in, we happened to go to a meeting where the Head of the MRC, the Medical Research Council, was giving a talk and he said that in the last few years the MRC had begun to really realise that they couldn't cure everything, that they couldn't cure the diseases that would be cured in the next millennium from a top down perspective. There had to be a trick, there had to be a bottom up as well, because that was the only way this was going to happen. And I have to say that that was a really reassuring moment in time for us to realise that we weren't just chasing pipe dreams and trying to do something impossible, that there was a role for us.    Ana Lisa: I think it would be really interesting for people to hear your story and the amazing set-up and fundraising that you've done, and at the same time it would be really good for us to reflect on how this isn't feasible for every patient and every family and how we're going to need to work cooperatively to move forwards with rare therapies.  Anne: When we explored the idea with Professor Muntoni and Meriel and others about setting up a charity one of the really reassuring things that Professor Muntoni got across to us was that this wasn't about raising the millions and millions it would take to fund clinical trials but the issue in the rare disease space was funding the proof of principle work, the work where you take a scientist's hypothesis and take it over the line, and the rarer the disease, the less places there are for a scientist to take those ideas. And the example he gave us was a piece of research like that might cost a hundred to a couple of hundred thousand, if you fund a piece of work like that and if it is successful, if the scientist's principle gets proven, then behind you it's much easier for the bigger muscle disease charities to also invest in it. It's harder for them to spread their money across all the very rare diseases hypothesis out there, but if you've helped a scientist get over the line they'll come in behind you and then they won't be the ones who fund the tens of millions that it takes to run a clinical trial.    If it's got potential, then that's where the commercial world comes in, and that's where the biotechs come in. So he'd given the example of if you spent £ten0,000 on a piece of research and it actually is proven, in behind you will come the bigger charities that would put in the million that takes it to the next phase, and in behind them will come the bio-checks that'll provide biotechs that'll provide the tens of millions.    And then, you know, a lot of what happens relies on serendipity as well, we know that, and you could easily run away with the idea that you made everything happen but you don't, you stand on the shoulders of others. And our very first grant application in our first grant round, which received extraordinary peer review for how excellent the application was, was a £100,000 project for a 3-year project that had gene therapy at the core of it by a researcher called Dr Ana Buj Bello at Généthon in Paris. This piece of research was so promising that 18 months in she and another researcher were able to raise $780,000 and, as Professor Muntoni predicted, from the French muscle disease charity AFM and the American muscle diseases charity MDA.  And 18 months into that 3 years it was so promising that a biotech company was started up with $30 million funding, literally just on her work.    So that doesn't always happen but, as Professor Muntoni explained, our job was not that $30 million, our job was that first £100,000, and our job was also to make ourselves known to the people in the neuromuscular field.  If you have lab time, if you have research time and you have a choice where you're putting it there is a place you can go to for a myotubular myopathy related grant application, so it's not just that this will come to us out of the blue, people will have done prior work, and our existence makes it worth their while, hopefully, to have done that prior work.  Ana Lisa: That's an amazing story how you've set up this charity and how successful that first application for gene therapy was. I'd love to hear more about that gene therapy and did it get to the clinic and to hear that story from you.  Because I think there are a lot of learnings and it's really important that the first patients who are treated, the first families that are involved, the researchers who start researching in this area, the first treatments lead the way and we learn for all the other treatments for all the other rare conditions that we hope and that together as a community we can share these learnings.  Anne: Yeah. I sometimes describe it a bit like going out into space. When you see a rocket going off look at how many people are behind and the amount of work that's been done, the degree of detail that's managed, and then you go out into space and there are a whole load of unknowns, and you can't account for all of them.  Who knows what's out there in this sphere.  But the amount of preparation, it feels similar to me now, looking back.  We were so idealistic at the beginning.  Our grant to Dr Buj Bello was 2008 and actually it is a really fast time in, the first child was dosed in the gene therapy trial in September 2017.  Ana Lisa: So, we're talking less than 1 years.  Anne: Yeah. And in the meantime obviously as a charity we're also funding other proof of principle research. One of the founding principles of the charity was to have a really excellent peer review process and scientific advisory board so that we wouldn't get carried away with excitement about one lab, one research team, that everything would always come back to peer review and would be looked at coldly, objectively. I don't know how many times I've sat in a scientific advisory board meeting with my fingers crossed hoping that a certain application would get through because it looked wonderful to me, and then the peer review comes back and there are things you just don't know as a patient organisation. So, yes, in those 9 years we were also funding other work.  Ana Lisa: You've just given an interesting perspective on sharing the learnings between the scientists, clinicians, the experts in a particular condition, if you like, and the families, and I'd be really interested to hear your views on what's been learnt about how families and the patient community can also teach the clinical and scientific community.  Anne: So, the first child was dosed in September 2017 and by the World Muscle Society Conference 2 years later in October 2019 the biotech had some fantastic results to show. Children who had been 24-hour ventilated were now ventilator-free, which, unless you know what it's like to have somebody in front of you who's ventilator-dependent, the idea that they could become ventilator-free is just extraordinary.    However, one of the things we've learnt about gene therapy is that we are going out into space so there are extraordinary things to be found, and extraordinary results are possible, as is evidenced here, but there is so much that we don't know once we are dealing with gene therapy. So unfortunately, in May, June and August of 2020, 3 little boys died on the clinical trial. So we have a clinical trial where the most extraordinary results are possible, and the worst results are possible, and both of those things are down to the gene…  What we discovered and what is still being uncovered and discovered is that myotubular myopathy is not just a neuromuscular disorder, it is a disorder of the liver too, and these children didn't die of an immune response, which is what everybody assumes is going to happen in these trials, they died of liver complications.    And one of the things that has come out of that, well, 2 sides to that. Number one is that it is extraordinary that we have found a treatment that makes every single muscle cell in the body pick up the protein that was missing and produce that protein, but also what we've understood is that the knowledge and experience of families and patients is even more vital than we've all been going on about for a long time. Because the issue of there being a liver complication in myotubular myopathy has been hiding in plain sight all this time, because if you asked any family they would tell you, “Yes, my son has had the odd liver result, yes.”    We could see something that looked like it was not that relevant because it was outside the big picture of the disease, which was about breathing and walking and muscles, but actually there was this thing going on at the same time where the children had liver complications. There were some very serious liver complications but everybody thought that was a minor issue but if we are able to engage the people who live with the disease and the people who observe the disease at a much more fundamental level we may be able to see more about what these rare genes are doing.  Ana Lisa: Yeah, thank you very much for sharing such a moving story and with such powerful lessons for the whole community about how we listen to the expertise that families have about their condition, and also I think the really important point about how we tackle the research funding so that we're including and sharing learnings from the conditions that are initially studied in greater depth, and we hope that many more conditions will be better understood and more treatments found and that actually the learnings from these first gene therapy trials will really help inform future trials, not just for gene therapies but also for many other novel therapies that are being developed.  [Music plays] If you're enjoying what you've heard today, and you'd like to hear some more great tales from the genomics coalface, why don't you join us on The Road to Genome podcast. Where our host Helen Bethel, chats to the professionals, experts and patients involved in genomics today. In our new series, Helen talks to a fantastic array of guests, including the rapping consultant, clinical geneticist, Professor Julian Barwell, about Fragile X syndrome, cancer genomics and a holistic approach to his practice - a genuine mic-drop of an interview. The Road to Genome is available wherever you get your podcasts. [Music plays] Ana Lisa: Carlo, I would really like to come to you about some of the initiatives that are happening in the UK, and particularly it would be really interesting to hear about the UK Platform for Nucleic Acid Therapies as a sort of shining example of trying to do something at a national scale across potentially many different rare conditions.    Carlo: Thanks, Ana-Lisa. Thanks very much, Anne, for sharing your fantastic story. I mean, I just want to iterate that as clinician scientists we do constantly learn from experiences and constantly learn from you, from the patient community, and this is absolutely valuable to push the boundary. And I really liked your vision of a rocket being launched in space and I would imagine that this is a similar situation here. So, we are facing a major challenge. So, there is over 7,000 rare diseases in the world and with improvements of genetic diagnosis this is only increasing. So, in a way rare diseases is the ultimate frontier of personalised medicine and this poses incredible challenges.   So, you mentioned the bottom-up approach and the top-down approach and in a way, both are absolutely necessary. So your story is a fantastic story but also makes me think of all the other families where they don't share perhaps the same spirit, you know, they are in areas of the world that are not as well connected or informed, where patient community simply cannot be ‘nucleated', let's say, around the family. So, there is definitely an issue of inclusivity and fair access.    So, what we're trying to do at UPNAT, which is the UK Platform for Nucleic Acid Therapy, is to try to streamline the development both at preclinical and clinical level of nucleic acid therapies. So, we'll start with antisense oligonucleotides just because those are the molecules of the class of drugs that are most ‘mature', let's say, in clinic. So, there are several antisense oligonucleotides already approved in the clinic, we know that they are reasonably safe, we understand them quite well, but of course the aspiration is to then progress into other forms of gene therapy, including gene editing approaches, for example.   And one of the activities that I'm involved, together with Professor Muntoni, is to try to streamline the regulatory process of such therapies and in particular curate a registry of, for example, side effects associated with nucleic acid therapy in the real world, and you would be surprised that this is something that is not yet available.  And the point is exactly that, it's trying to understand and learn from previous mistakes perhaps or previous experiences more in general.    And this is very much in synergy with other activities in the UK in the rare disease domain.  I'm thinking of the Rare Disease Therapy Launchpad, I'm thinking of the Oxford Harrington Centre, I am thinking of the recently funded MRC CoRE in Therapeutic Genomics. These are all very synergistic. Our point is we want to try to amplify the voice of the patient, the voice of the clinicians working on rare disease, and we want to systematise. Because of course one of the risks of rare disease therapies is the fragmentation that we do all these things in isolation. And I would argue that the UK at the moment leveraging on the relatively flexible and independent regulatory agencies, such as the MHRA, on the enormous amount of genetics data available through Genomics England, and of course the centralised healthcare system, such as the NHS, is really probably the best place in the world to do research in the rare disease area, and probably I'm allowed to say it because I'm a non-UK native.       Ana Lisa: Thank you, that's a brilliant perspective, Carlo, and across all the different therapeutic initiatives that you're involved with. And, Carlo, presumably - we're all hoping - these different initiatives will actually lead to ultimately a bigger scaling as more and more novel therapies that target both our RNA and DNA and actually are working, I guess further upstream in the pathway.    So classically in the past it's been necessary to work out all the underlying biology, find a druggable target somewhere in that pathway and then get a larger enough clinical trial, which can be nearly impossible with many of the rare and ultra-rare conditions or even, as you've said, the sub-setting down of more common condition into rarer subtypes that perhaps can be treated in different ways.  And with the many new different treatments on the horizon, ASO therapies, as you've said, is a place that's rapidly expanding, and also crisper gene editing. I'd be really interested to hear your reflections on how this might scale and also how it might extend to other new treatments.  Carlo: Yeah, that's exactly the right word, ‘scaling up'. I mean, there will be of course very unique challenges to every single rare disease but I would argue that with genetic therapies, such as ASOs or crisper gene editing, the amount of functional work that you need to do in a lab to prove yourself and the scientific community that this is the right approach to go for can be certainly very important but can be less just because you're addressing very directly because of the disease.    And then there are commonalities to all these approaches and possibly, you know, a platform approach type of regulatory approval might serve in that regard. You know, if you are using the same chemistry of these antisense oligonucleotides and, you know, similar doses, in a way the amount of work that you need to produce to again make sure that the approach is indeed a safe approach and an effective approach might be also reduced.    I would say that there are also challenges on other aspects of course, as you were saying, Ana-Lisa. Certainly the typical or standard randomised placebo control trial that is the standard and ultimate trial that we use in a clinical setting to prove that a molecule is better than a placebo is many times in the context of rare diseases simply not possible, so we need to think of other ways to prove that a drug is safe and is effective.   This is something that we all collectively as a scientific community are trying to address, and the alliance with the regulatory agencies, such as the MHRA, and you said that you have found your interaction with the MHRA very positive, and I can tell you exactly the same. So we are all trying to go for the same goal, effectively, so trying to find a way to systematise, platformise these sort of approaches. And I guess starting with antisense oligonucleotides is really the right place to go because it's a class of drugs that we have known for a long time, and we know it can work.  Ana Lisa: Meriel, can you tell us a little about the National Genomic Research Library at Genomics England and how this could link with initiatives to find many more patients as new treatments become available for rare and ultra-rare conditions?  Meriel: Yes, I think what's wonderful now is actually that what we're really trying to do is give everybody the opportunity to have their rare condition specifically diagnosed at the molecular level, and the way in which that is being done is by offering whole genome sequencing in the NHS currently in England but to all patients with rare diseases.    And so, it's about trying to establish their diagnosis. And as well as that, even if the diagnosis isn't definitely made at the first pass when the clinical scientists look at the data, because the whole genome has been sequenced, actually all that information about their genome, if they consent, can then be put into the National Genomics Research Library.  And that is a fantastic resource for national and international researchers who get approved to work in this trusted research environment to make new disease gene discoveries and identify these diagnoses for patients.  What's also offered by Genomics England as well is when the National Genomics Library data results in a new publication, the discovery of a new gene or perhaps a new molecular mechanism that causes a disease we already know about, that feeds back into the diagnostic discovery pathway within Genomics England back onto the diagnostic side of all the data.    So, patients who may have had genetic testing previously using whole genome sequencing where they've, if you like, had their sequencing done before the diagnosis was sort of known about, will also be picked up. And so, what this is really doing is trying to kind of give this really equal platform for everybody having testing to all have the same opportunity to have their diagnosis made, either on the diagnostic side or with research.  Ana Lisa: So, sort of on a cohort-wide scale as new discoveries are made and published you can go back and find those patients that may actually have that diagnosis and get it back to them, which is brilliant.  Meriel: Exactly. And this speeds up the whole process of getting these diagnoses back to people. So on a regular basis in the NHS, we will get feedback from the Diagnostic Discovery Pathway about “Here's some patients who you requested whole genome sequencing from a number of years ago and actually now we think we know what the particular molecular condition is.”  And so, it's key of course for our patients with rare conditions to make that molecular diagnosis because then we're able to have them identified for our colleagues who are doing this ground-breaking research trying to bring therapies for these rare conditions.  Ana Lisa: Thank you. And I hope that, as currently, if a novel genetic mechanism, as you've just described, is identified that could explain a rare condition that those patients can be found and they can receive that diagnosis, even many years later, and hopefully as novel treatments become available and say there's a chance to individualise ASO therapies, for example, to start with, that one could also go and look for patients with particular variants that could be amenable potentially to that treatment. And that's really sort of exciting that one could look for those patients across England, irrespective of which clinic they're under, which specialist they're under, and I think that could be really powerful as new treatments develop. I suppose, Meriel, if somebody comes to see you now in clinic are things different?  Meriel: Well, I think one of the things for me when patients come to clinic now is we might have an idea about what we think their condition is, maybe even we think it's a specific gene. And we can offer whole genome sequencing and so it's not just the way we used to do things before by looking just at the coding regions of the gene, we can find more unusual ways in which the gene can be perturbed using whole genome sequencing.  But let's say we don't make the diagnosis. I encourage my patients, if they're comfortable with it, to join the National Genomics Research Library, because really it's been incredibly productive seeing the new genetic discoveries that are coming out of that, but as well I say to them, even if we don't get the diagnosis the first time round when we look at the data, actually this is a constant cycle of relooking at their data, either if they're in the NGRL or as well on the Diagnostic Discovery Pathway side of the service that's run by Genomics England. So yeah, I feel like it's a very big difference; they don't have to keep coming every year and saying, “Is there a new test?” because actually they've had an excellent test, it's just developing our skills to really analyse it well.  Ana Lisa: Yes, and our knowledge, the technology and the skills keep evolving, certainly.  And I think one of the things that I'm sort of hearing from this conversation is that balance of hope and realism, Carlo we were talking about earlier how you need all the pieces of the puzzle to be lined up - so the regulatory agency, the clinicians, all the preclinical work has to have been done, monitoring afterwards for side effects - every piece of the puzzle has to be lined up for a new treatment to make it to a patient.    And, Anne, I'd like to come back to you because we've talked about this before, how one balances these messages of optimism and hope which are needed for bringing everybody together as a community to crack some of these very difficult challenges highlighted by treatments for rare and ultra-rare conditions and at the same time the need for realism, a balance conversation.  Anne: Yeah, that was one of our big learnings through the gene therapy trial and other trials we've had in the condition. As a rare disease charity, you do everything. You know, my title is CEO, but I tell people that's Chief Everything Officer because there's only a few of you and you do everything. So, you go and you lead the London Hope Walk and you also are a layperson on the Scientific Advisory Board and you also send out the emails about grants... And so, you could easily as a small rare disease charity conflate different communication messages because you're in a certain mode.  And so we have been from the early days in the mode of raising hope for people to say, “Look, we can make a difference as a patient community, we could raise funds, we might be able to move things forward, you've got the power to make a difference if you want to.” That's one set of hope.  And it's not dreamlike hope, we're linked to the reality of there are great breakthroughs.  So, you know, in the world of spinal muscular atrophy these clinical trials have led somewhere very quickly, so we're not selling false hope, we're talking about the difference we can make.    But then as soon as you flip into “There's a clinical trial being run” that's a completely different type of communication and you cannot conflate that message with the previous message.  And we always say to everybody, “We're your team, we're a family, we're a team, we all help each other.  When you are considering joining a clinical trial your team is the clinical trial team.    The other team does other things for you but the people you need to work with and ask hard questions of and listen hard to, that's your clinical trial team led by the principal investigator because then you're in that with them. And, you know, the reality of the fact that many, many clinical trials don't work as we wish they would be and the decision you make for your child, your baby, your little one, to join a clinical trial… because that's what it comes down to in our disease, has to be made with that team, not the team that's selling you a fundraising event. It's worth reminding rare disease patient organisations we're wearing different hats and the hope and the realism are different tracks you have to go down.    But at the same time as being realistic you also have to keep remembering that there is still grounds for hope, we are moving forward. And 21 years ago, when Tom was born the idea that you would be able to get all of the muscles in the body to switch back on – putting it in lay terms – seemed like a bit dream. Well, that is what has happened in the gene therapy clinical trial, we just have to now make it safer and understand more about what we're dealing with. So, the 2 things, the hope and the realism, do exist side by side.  Ana Lisa: I think that perfectly encapsulates a lot of the messages around rare disease therapies where there's such hope that novel treatments will really target directly the DNA or RNA to potentially correct the problem across many different rare conditions and therefore actually making treatments one day suddenly available to a much, much bigger population of people with rare conditions than we could've dreamt of 20 years ago or perhaps now, and at the same time this massive need to work cooperatively to all make this as fair, as equitable. Not everybody is going to have the opportunity to fundraise massively to be an expert about their condition, and the importance of sharing these learnings and also really, really listening to the patient community and really, as Carlo was saying, keeping track of side effects, having registries/databases to share these is going to be incredibly important.  [Music plays]  Ana Lisa:  Anne, can you tell us a little about your reflections on equity from the patient community perspective?  Anne: Well I mentioned serendipity early and one of the aspects of serendipity that played into our favour for setting up the Myotubular Trust was that by hook or by crook Wendy Hughes, who set up the charity with me, and I were both able to devote time at that period of our lives to setting up a charity. When my husband, Andrew, and I were told that Tom would more than likely die before his first birthday, one of the decisions we made as a family was that he would never not be with a parent, we would always have someone around, and that kind of meant someone had to give up a full-time job and that was me.  We thought, “If Tom has a few scarce months on the planet, we'll be with him.” And then when Tom lived to be nearly 4, as a family we got used to living on one salary and we were very lucky that we could pay the mortgage that way and run our family that way and eventually that meant I had the time to run the charity.    That doesn't happen that easily, that's a tall order, particularly when you have somebody in the family who has such high needs. And one of the things that I have often thought about is that in the rare disease space we could do with a different funding model for rare disease charities, we could, in an ideal world I have this nirvana that I imagine where there's a fund that you can apply to that is contributed to by the people who make profits out of finding rare disease cures - so the pharmaceutical companies and the biotechs - and there's a fund that they contribute to and that if you have a rare disease and you are willing to set up an organisation that supports families, that raises research funds, that provides a way of hearing the patient voice, then you could apply to that for running cost funds and then you'd be able to run this charity. And then you wouldn't have to rely on whether you live in an area where people will raise money for you or…  We were very lucky that we came across a few great benefactors who would give us money for running the charity, which is actually how we fund it.    All the research money we raise goes 100% into research, not a penny of it goes towards running costs because we have serendipitously found people who will be benefactors for the charity, but we're relying on a lot of good luck for that kind of model to work. And when you look at how much profit is made from developing rare disease treatments and cures – which is fine because that's what puts the passion and that gets people working on it – then why not have an advance fund to run rare disease charities? One of my nirvana dreams.  Ana Lisa: It's good to dream. Indeed, my hope is that there will be some amazing shining examples that lead the way that open doors, make things possible, prove that something can work and how and that then that will enable many other treatments for many additional rare conditions to be added in so that if you've learnt how this particular treatment modality works for this rare condition and there was funding behind it and everything else that's needed that then you can, the learning from that, I'm going to use the word ‘tweak', which sounds minor and could be very major but actually the concept that you can then tweak all those learnings and findings so that that same type of treatment modality could be adapted to treat somebody else with a different rare condition in a different location would be absolutely incredible and really powerful, given that if something like 85% of rare conditions affect less than one in a million people it's not going to be feasible to use the same strategies that have been used in the past for very common conditions.    One of the other big barriers is the cost of developing treatment for ultra-rare conditions.  Where it's a small number of patients that you have and therefore all the challenges that come with monitoring, checking for efficacy, monitoring safety and ultimately funding the challenges are much greater, however if some of these treatment modalities are also going to be used to treat common conditions it might be that actually there's a lot more cross-talk between the nano-rare, ultra-rare, rare and common conditions and that we can share a lot of that learning. I'd love to hear from each of you where you hope we will be for rare disease and rare therapies.  Carlo: Well my dream is that in 5 to 10 years' time an individual with a rare disease is identified in the clinic, perhaps even before symptoms have manifested, and at that exact time the day of the diagnosis becomes also a day of hope in a way where immediately the researcher, the centre, genetics lab, flags that there are the specific mutations, we know exactly which is the best genetic therapy to go after, antisense oligonucleotides as opposed to CRISPR editing, and a path forward, both at the preclinical and clinical level, to demonstrate and to cure these patients eventually is already laid out in front of the patient.  So, transforming the day of their diagnosis as a day of hope, this is my dream with the next ten years.  Ana Lisa: Thank you, that's a wonderful dream. Meriel, can I come to you?  Meriel: Yes, I think I just want to echo Carlo.  We've had great developments and progress with getting whole genome sequencing into the NHS for testing but what we really need is for it to be fast and efficient and getting those diagnoses established quickly. And we have had that set up now and we're really getting there in terms of speed, but then what we need is exactly what's the next step and actually structure like UPNAT that are developing these processes that we can then say to the patient, “And from there, now that we've established your diagnosis, this is what we have options to offer.”  Ana Lisa: Brilliant. And presumably that if the diagnosis isn't achieved now there is a hope that it will be achieved in the future as well. Anne...  Anne: Well, stepping one hundred per cent into the patient's shoes rather than the scientific side that we don't so much influence....  stepping in the patient's shoes, in 5 years' time I would absolutely love it if we were in a situation where all the parties that have come to the table looking at a therapy or in the earlier research genuinely want to bring the patient voice into the room. As Carlo talked about, there's even going to be more and more and more of these rare diseases, then those voices, those few people who have experience of it, they may be able to shed light on something. Maybe even sometimes don't even know it's a fact that they know but that were brought to the table as passionately as everything else is brought to the table.  [Music plays]  Ana Lisa: We'll wrap up there. Thank you so much to our guests, Anne Lennox, Carlo Rinaldi and Meriel McEntagart, for joining me today as we discuss the collaborative power of working together and look to the future of rare therapies that could have the potential to unlock treatments for many rare conditions. If you'd like to hear more like this, please subscribe to Behind the Genes on your favourite podcast app.  Thank you for listening.  I've been your host, Ana-Lisa Tavares. This podcast was edited by Bill Griffin at Ventoux Digital and produced by Naimah Callachand.  

In this final episode of our mini-series on the AstraZeneca Covid vaccine, Investigations Editor Claire Newell explores whether the MHRA, the regulatory agency for drugs, has protected patients. She hears from families about the long-term consequences of a rare adverse reaction to the jab, and whether they have received enough support from the Government.Listen to the first two episodes of The Daily T Investigates: The AstraZeneca vaccine hereListen to 'The Lockdown Files: The Forgotten Victims' hereWritten by: Claire NewellProducer: Jack BoswellExecutive Producer: Adélie Pojzman-Pontay Hosted on Acast. See acast.com/privacy for more information.

The treatment of Geographic Atrophy (GA) or late stage dry age-related macular degeneration as it's also known, is proving to be a pretty tough nut to crack. Hopes had been high that a treatment available in the USA would also be approved for use here in the UK. However, the Medicines and Healthcare products Regulatory Agency (MHRA) has rejected the application. Ed Holloway, Chief Executive of the Macular Society and Bill Best who has lived with GA for many years join us to discuss the MHRA decision.Optomap is an imaging system which produces significantly more detailed information about the retina than had been available before. This can lead to earlier diagnosis of many eye conditions and accordingly a better chance of preventing sight loss. Dr Peter Hampson, Policy and Clinical Director of the Association of Optometrists and John Hopcroft, Clinical Services Manager at Boots join us to discuss the system and how public access to it is being improved by bringing it to the high street. Presenter: Peter White Producer: Fern Lulham Production Coordinator: David BaguleyWebsite image description: Peter White sits smiling in the centre of the image, wearing a dark green jumper. Above Peter's head is the BBC logo (three individual white squares house each of the three letters). Bottom centre and overlaying the image are the words "In Touch"; and the Radio 4 logo (the word Radio in a bold white font, with the number 4 inside a white circle). The background is a bright mid-blue with two rectangles angled diagonally to the right. Both are behind Peter, one of a darker blue and the other is a lighter blue.'

 HO HO HO, MERRY FISMAS!!!On the Third & Final Pod of FISmas these ID:IOTS have for you… 6 golden topics! Listen to hear about:1. Personalised approach to invasive fungal infection2. Agricultural anti-fungals and One Health3. Candida auris4. NTM standards of care5. Narrative reporting 6. Fluoroquinolones and the MHRA warnings Jame and Callum attended the Federation of Infection Societies 2024 annual meeting in  Liverpool; here they share their reflections on the sessions  they attended on:  a wide mix of topicsWe hope these episodes have been ADVENTatious to your learning.Notes for this episode here Send us a text Support the showQuestions, comments, suggestions to idiotspodcasting@gmail.com or on X/Threads @IDiots_podPrep notes for completed episodes can be found here (Not all episodes have prep notes).If you are enjoying the podcast please leave a review on your preferred podcast app!Feel like giving back? Donations of caffeine gratefully received!https://www.buymeacoffee.com/idiotspod

FREEDOM - LIBERTY - HAPPINESS SUPPORT DOC MALIK To make sure you don't miss any episodes, please subscribe to either: The paid Spotify subscription here: ⁠https://podcasters.spotify.com/pod/show/docmalik/subscribe The paid Substack subscription here: ⁠https://docmalik.substack.com/subscribe Thank you to all the new subscribers for your lovely messages and reviews! And a big thanks to my existing subscribers for sticking with me and supporting the show! ABOUT THIS CONVERSATION: In this chat, I sit down with Dr Liz Evans to discuss why we need a medical freedom movement here in the UK, especially after everything we've seen during the COVID-19 pandemic. We talk about the ethical concerns around vaccine safety and informed consent. Liz opens up about her background as a former NHS doctor, her shift into complementary medicine, and how her Christian faith plays a huge role in her commitment to medical ethics. We also discuss how the UK Medical Freedom Alliance came to be and the worrying changes in public health legislation (Scotland, Canada, and now Northern Ireland) that could lead to government overreach. The conversation highlights why public resistance is so important and why we all need to take a stand. Ultimately, it's about fighting for a society that respects individual freedoms and upholds medical ethics. Ahmad x Links Website UK Medical Freedom Alliance X UKMFA X Proposed Public Health Bill Consultation Link UKMFA Open letter to JCVI, MHRA and Matt Hancock, in Nov 2020, urging them not to authorise or roll out the Covid vaccines due to serious safety & ethical concerns IMPORTANT INFORMATION AFFILIATE CODES Hunter & Gather Foods ⁠Hunter & Gather Foods Use DOCHG to get 10% OFF your purchase with Hunter & Gather Foods. IMPORTANT NOTICE Following my cancellation for standing up for medical ethics and freedom, my surgical career has been ruined. I am now totally dependent on the support of my listeners, YOU. If you value my podcasts, please support the show so that I can continue to speak up by choosing one or both of the following options - ⁠Buy me a coffee⁠ If you want to make a one-off donation. Join my Substack To access additional content, you can upgrade to paid from just £5.50 a month Doc Malik Merch Store⁠ Check out my amazing freedom merch To sponsor the Doc Malik Podcast contact us at ⁠hello@docmalik.com⁠ Check out my website, visit ⁠www.docmalik.com

Justice for Reyzl: Standing up to LGBTQ Workplace Discrimination Host: Noah Parrish, Gender Justice Communications Director Guest: Brittany Stewart, Gender Justice Senior Staff Attorney In August 2024, Gender Justice and co-counsel Wanta Thome PLC filed a discrimination lawsuit against the Academy of Holy Angels, a Catholic high school in Richfield, Minnesota, and the Archdiocese of St. Paul and Minneapolis after they effectively terminated Reyzl Grace, a staff librarian, when she came out as trans. Reyzl Grace's story highlights the importance of ensuring that no employer in Minnesota has carte blanche to discriminate against employees simply because of who they are.   We believe the school violated the Minnesota Human Rights Act (MHRA) by refusing to allow Grace, who served as a secular librarian at the school, to reapply for her position on the basis of gender identity and sex. While the MHRA does permit limited exemptions on religious grounds, the Legislature never intended for these exemptions to apply to secular employees. In this episode of The Gender Justice Brief, Reyzl Grace's attorney, Brittany Stewart, outlines our case seeking justice for her: Background and context on Reyzl Grace's case Grace v. Academy of Holy Angels — Fact Sheet Read the complaint here Sign up to stay informed on Reyzl Grace's case ### Visit the "Gender Justice" Website ⁠here⁠ and "Unrestrict Minnesota" ⁠here⁠. The GJB is produced by Michael at ⁠www.501MediaGroup.com⁠ & Audra Grigus.

The Medicines and Healthcare products Regulatory Agency better known as the MHRA, rearranges to spells HARM and they now prefer to be Enablers not Regulators.

On this episode, Dr. Tala Fakhouri (Associate Director for Data Science and Artificial Intelligence Policy, FDA) and Stephen Pyke (Chief Clinical Data & Digital Officer, Parexel) join the podcast to discuss how the FDA and regulators around the world are thinking about the use of AI in clinical research.They dive deeper into the FDA's evidentiary standards for AI, what organizations should consider about methodological transparency in submissions, and AI's potential as a tool that can help bring medicines to market more efficiently.For more information on ACRO's AI/ML Principles Statement, visit ACRO's website: https://www.acrohealth.org/initiatives-hub/ai-ml-in-drug-development/

As AstraZeneca forges on creating more ‘vaccines', what about those left behind who have lost everything? Blocked on social media by the UK's medicines regulator and reported to a government agency for ‘misinformation', Adam tells what life is like when you are vaccine-injured or bereaved. Read the write-up at: https://www.ukcolumn.org/video/blocked-by-the-mhra-reported-to-the-health-security-agency-a-day-in-the-life-of-vaccine

Pink Sheet reporter and editors discuss what an upcoming listening session means for the FDA's advisory committee reform effort (:32), the agency's efforts to help the clinical trial modeling and simulation industry (16:22), and the UK's MHRA plan to use artificial intelligence to assist in drug application reviews (21:05). More On These Topics From The Pink Sheet US FDA Adcomm Reform: Does Listening Session Suggest No Major Near-Term Changes?: https://pink.citeline.com/PS150167/US-FDA-Adcomm-Reform-Does-Listening-Session-Suggest-No-Major-Near-Term-Changes US FDA Wants Advice About Advisory Committees: Try Having Some?: https://pink.citeline.com/PS154644/US-FDA-Wants-Advice-About-Advisory-Committees-Try-Having-Some US FDA Developing Model Master File System To Grow Modeling, Simulation Field: https://pink.citeline.com/PS154647/US-FDA-Developing-Model-Master-File-System-To-Grow-Modeling-Simulation-Field UK's MHRA To Use AI In Regulatory Review Process & RWD Analysis: https://pink.citeline.com/PS154643/UKs-MHRA-To-Use-AI-In-Regulatory-Review-Process--RWD-Analysis

How many medicines can you think of created for just one person? The likelihood is none - which is why the world hasn't heard of milasen yet. But its creation, and the efforts behind it, could build a pathway towards some of the greatest advances in genomic medicine, and a new initiative being trialled in Britain has a huge role to play in making this happen. At the age of seven, Mila Makovec became the first person in the world to be treated with a medicine created just for her. A bubbly young girl from Colorado, Mila suffered from a very rare genetic disorder called Batten disease, which leads to a painful early death in children. Mila's mother, Julia Vitarello, resolutely sought out scientists to try to discover a way to save her daughter. After relentless efforts, one doctor, Timothy Yu from Boston Children's Hospital, imagined a possible treatment for Mila. The challenge was it involved making a completely unique treatment for Mila's specific genetic mutation. It would be novel and very expensive - but it was her only option. Julia raised the millions of dollars required through a charity she set up in her daughter's name, and in 2018 Mila became the one patient in the world to receive the drug milasen. Initially, it worked, and Mila's condition stabilised and improved. However, the treatment was given after the disease had done a great deal of damage to a small child, and Mila died when she was ten years old.There are an estimated 7,000 rare diseases in the world, affecting more than 400 million people - and most are genetic. The majority have no effective treatment. New medicines for these conditions can't be put through clinical trials on groups of patients because they are so rare. So, currently, such novel therapeutics can only be legally given after lengthy and costly work that is uncommercial for drug firms. Having got so achingly close to saving her daughter, Mila's mother is now leading efforts to make these new genetic medicines available to other children with rare diseases - and Britain is where her campaign is about to take a huge step forward. The launch of the Rare Therapies Launch Pad is bringing together efforts from Mila's Miracle Foundation, the UK medicine's regulator the MHRA, Genomics England and Oxford University in an world leading attempt to build a new streamlined regulatory pathway to allow one-off drugs to be designed and approved for use in individual patients with rare diseases. Natasha Loder, Health Editor at the Economist, tells this very personal story of how one mother's determination to try and save her daughter could lead to a revolution in personalised medicine - one that has the potential to bring hope to millions of families. Producer: Sandra Kanthal