Podcasts about therapies

While endometriosis is highly associated with Chronic Pelvic Pian (CPP), some women may suffer from a different primary or coexistent secondary etiology: pelvic vascular congestion, called vascular origin (VO)- CPP. Although controversial as an entity, there have been diagnostic algorithms published (via pelvic ultrasound. MRI, or venography) for this condition. Approximately 10-40% of chronic pelvic pain cases may be attributed to pelvic vascular congestion (now termed pelvic venous disorder), though estimates vary considerably depending on the population studied and diagnostic criteria used. In premenopausal women specifically, the prevalence appears higher. One study found that 8% of all premenopausal women had documented chronic pelvic pain of unclear etiology along with dilated ovarian and pelvic veins on cross-sectional imaging. Therapies for this have been limited. Flavonoids are abundant in a colorful diet of fruits, vegetables, tea, and wine, with common sources including citrus fruits (flavanones), berries, apples, grapes (flavan-3-ols/anthocyanins), onions, kale, broccoli (flavonols), and tea, cocoa, red wine (flavan-3-ols), plus soybeans (isoflavones), all providing antioxidants and potential health benefits like better heart and brain health. On Dec. 23, 2025, in the journal Phlebology, researchers published a systematic review on the potential benefits of specific flavonoid mixtures which may provide relief to VO-CPP. Listen in for insights and details.1. Gloviczki ML, Demetres MR, Salazar G, Khilnani NM. Venoactive drugs for venous origin chronic pelvic pain in women: A systematic review. Phlebology. 2025 Dec 23:2683555251411027. doi: 10.1177/02683555251411027. Epub ahead of print. PMID: 41432346.2. Knuttinen MG, Machan L, Khilnani NM, Louie M, Caridi TM, Gupta R, Winokur RS. Diagnosis and Management of Pelvic Venous Disorders: AJR Expert Panel Narrative Review. AJR Am J Roentgenol. 2023 Nov;221(5):565-574. doi: 10.2214/AJR.22.28796. Epub 2023 Apr 5. PMID: 37095667.

Drs. Herzberg and Yu continue their discussion on emerging clinical data presented at ESMO and WCLC 2025. They highlight recent advancements in HER2-targeted therapies for NSCLC and review new HER2-targeted therapies, international study results, and the promise of evolving targeted approaches for HER2-altered lung cancer.

Send us a textAs 2025 comes to a close, Ben and Daphna reflect on a year of growth, community, and evolution for The Incubator Podcast. In this end-of-year wrap-up, they preview major changes coming in 2026, including new standalone podcast feeds, expanded journal club content, CME opportunities, and exciting partnerships with organizations like the Vermont Oxford Network and PAS. They also share what's ahead for the Delphi Conference and offer a candid look at their personal and professional goals for the year ahead. Thank you for being part of this extraordinary neonatal community. Support the showAs always, feel free to send us questions, comments, or suggestions to our email: nicupodcast@gmail.com. You can also contact the show through Instagram or Twitter, @nicupodcast. Or contact Ben and Daphna directly via their Twitter profiles: @drnicu and @doctordaphnamd. The papers discussed in today's episode are listed and timestamped on the webpage linked below. Enjoy!

Drs. Herzberg and Yu explore emerging clinical data from the 2025 meetings of the European Society For Medical Oncology (ESMO) and World Conference on Lung Cancer (WCLC), highlighting recent advancements in HER2-targeted therapies for NSCLC. Their discussion focuses on new drugs (eg, zongertinib and trastuzumab deruxtecan), their efficacy and safety profiles, and the potential for treating HER2 mutations and overexpression.

Send us a textTransitioning from fellowship to your first attending job? You're probably not prepared for the business side of medicine—and that's exactly the problem Dr. Tung Giep addresses in this episode. Dr. Giep, a neonatologist with over 30 years of experience, shares hard-earned lessons from building and selling a private practice in Houston, navigating toxic work environments, and eventually finding his place in telemedicine. His new book, The Business of Medicine: The Definitive Guide to Help New Physicians Start Their Career on the Right Path and Avoid Costly Mistakes, tackles what medical training ignores: contract negotiation, non-competes, choosing the right CPA and attorney, and understanding your self-worth.Ben and Dr. Giep discuss why new physicians get blindsided by job offers, how to vet a practice properly, and the reality of community neonatology versus academic medicine. They also explore the growing role of AI in healthcare and what it means for the next generation of physicians. Whether you're finishing fellowship or reconsidering your current position, this conversation offers practical guidance on taking control of your career—and avoiding costly mistakes along the way. Support the showAs always, feel free to send us questions, comments, or suggestions to our email: nicupodcast@gmail.com. You can also contact the show through Instagram or Twitter, @nicupodcast. Or contact Ben and Daphna directly via their Twitter profiles: @drnicu and @doctordaphnamd. The papers discussed in today's episode are listed and timestamped on the webpage linked below. Enjoy!

Join us in this episode with expert guest Dr. Mahvish Rahim (the Children's Hospital at Montefiore) as we dive into the rapidly evolving world of curative and transformative therapies for sickle cell disease! From breaking down the science behind stem cell transplant and gene therapy to understanding how to choose the right candidates and counsel families, Dr. Rahim brings clarity to this exciting new era in SCD care.

Our series concludes with Cathy Carson sharing about energy medicine, and specifically, things that you could do for self or others.

  In this third and final deep-dive episode, Nicholas Simmonds highlights the importance of collaborative networks, such as the European Cystic Fibrosis Society (ECFS) Clinical Trials Network, in enabling global clinical trials. He also emphasises the central role of the patient voice in developing new therapies, using CFTR modulators as a key example. Timestamps: 00:00 – Introduction 00:55 – ECFS Network 03:29 – Trial design 04:54 – Therapy frontiers 07:48 – Curative approaches 13:07 – Clinician message

Alarming in immunoglobulin A nephropathy (IgAN): over 40% present late. Explore latest advancements in management of the most common cause of primary glomerulonephritis worldwide. Credit available for this activity expires: 12/23/26 Earn Credit / Learning Objectives & Disclosures: https://www.medscape.org/viewarticle/navigating-challenges-igan-management-clinical-implications-2025a1000w9b?ecd=bdc_podcast_libsyn_mscpedu

In this episode of Off Script, we continue our conversation with Jason Bock, co-founder and CEO of CTMC, diving deeper into the operational and manufacturing challenges shaping the future of curative cell therapies. Jason discusses how CTMC—through its partnership with MD Anderson Cancer Center—is eliminating inefficiencies across development timelines to significantly accelerate clinical progress without increasing risk. He also shares a forward-looking perspective on how cell therapies could one day be ordered and delivered like traditional pharmaceuticals, and what scientific, regulatory, and manufacturing innovations will be required to make that vision a reality.

Julie A. Parsons, MDHaberfield Endowed Chair in Pediatric Neuromuscular DisordersProfessor of Clinical Pediatrics and NeurologyUniversity of Colorado School of Medicine, Children's Hospital ColoradoAurora, CO, USAThe gene transfer trials for musculoskeletal disorders, if we look at musculoskeletal and neurologic disorders, we really do have the highest success rate in terms of treatment, but we also carry the highest incidence of treatment-emergent severe adverse events. And why is that true? Yesterday, when we were hearing about Donovan as well, we looked and said, When the first gene transfer therapies were started, he had a single muscle that was injected.When we look at Luxturna, we injected the retina. Now, what is happening with these disorders is that we're giving these huge, massive doses of viral vector to patients. There haven't been a lot of gene transfer therapies that have reached the market. But you saw yesterday, so many gene transfer therapies being worked on, but there are very few that have actually come to market. There are a couple of reasons for that.One is with the indications that we have, we know that the musculoskeletal disorders are most likely to achieve benefit, but there are the high risk of severe adverse events. Route of Administration, IV, for most of our disorders is the way we're going. We may end up having some Intrathecal therapies as well that are coming on board, but right now it's IV, and that means, a huge dose of this viral vector and antigenic risk that is being administered.In the vector design now, we actually have more specific vectors as well as promoters that are being utilized to really target specific tissues, so that we're able to focus in a little bit more on the tissues that we want to have affected. And then the dose has gone from these little tiny local injections to really systemic, much broader. And now our patients, are larger. So we're giving a viral genome per kilo dose that is just massive as we look at that.Then there really are challenges in terms of the translation of clinical trials to commercial treatment with these agents. And we don't always know, we're not always great when we do tests in clinical trials in small populations, about when that's broadened to the commercial availability and we hit larger heterogeneous populations.There are safety issues arising from these therapies, and I think that we have some experience now, certainly with the three diseases that I mentioned at the beginning, in terms of collecting some data and information to have a little bit more of an idea what to expect. Although to me, the recurring esteem is always, expect the unexpected. Because we still are learning about this. Hepatotoxicity. We know that transaminitis is something that we see in almost every gene transfer therapy that has been delivered, and we have to watch really, really closely and follow our patients closely for this. We also have to select patients that we don't think have risk for additional liver injury or underlying liver pathology, because as we found out in the XLMTM boys, we missed that. Thrombotic Microangiopathy. We look at this disorder. We've had deaths in SMA from TMA. We have Duchenne patients that have had TMA.This is scary because as many of us as clinicians who have treated patients, you know that we end up getting thrombocytopenia. So is that it this time, or are they going to be fine, or the platelet is going to go back to normal? This is another one that we have to watch really, really closely for. Cardiac Toxicity. We have had cardio myositis. We've had deaths from cardiac toxicity.Something really, really important for us to think about. In little kids, vomiting could be a sign of cardiac myositis. And for most of us who've treated patients with gene transfer therapy, what's one of the first issues that you get?You get nausea of vomiting, they don't feel good. So is that myocarditis or is it just a standard side effect that we're seeing with treatment? Importantly, as we discovered, there actually can be an immune response to the transgene. It's not just the viral vector capsid, it's actually the transgene as well. That was discovered in patients who were treated for Duchenne. So that's a really important thing in terms of looking now at what's our patient's selection and how do we pick the right patients.Next part, Dr. Parsons will discuss understanding and preparing risk factors associated with AAV gene therapies.

Alan Beggs, PhDDirector of the Manton Center for Orphan Disease ResearchSir Edwin and Lady Manton Professor of Pediatrics, Boston Children's HospitalHarvard Medical School, Boston, MA, USA Julie A. Parsons, MDHaberfield Endowed Chair in Pediatric Neuromuscular DisordersProfessor of Clinical Pediatrics and NeurologyUniversity of Colorado School of Medicine, Children's Hospital ColoradoAurora, CO, USADoctors Beggs and Parsons discuss the current status of gene therapies in rare neuromuscular disorders in this eight part podcast series. This is derived from the symposium that was presented at the MDA 2025 conference in Dallas, Texas, in March 2025 and is intended for healthcare professionals only. This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established. In contents of this podcast, shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The ASPIRO clinical trial is on clinical hold since September 2021.In this part, Doctor Beggs will provide an explanation of AAV-mediated gene therapies.Alan Beggs, PhDAAV vectors, which I'm going to be talking about more today, or Adeno associated viral vectors are small viruses. Their DNA gets delivered into the cell and remains extrachromosomal. There are very rare occasional integrations, but the risk of oncogenesis as a result is significantly lower as a consequence of remaining extrachromosomal, though, we do have to think about what happens as the cells divide and potentially the durability of treatment is more limited.There have been a lot of movement and development over the years, starting back in the 1980s when the first AAV genomes were isolated and sequenced. This led to a development of methods to produce recombinant AAVs that would lack the genes necessary for viral replication, but contain a therapeutic gene you wish to deliver. Through this, the structure of AAVs have been developed. There have been isolation of a number of naturally occurring variants. You've heard of AAV8, AAV9, also RH 74, derived from a rhesus monkey for the RH. These have all been used in clinical trials. Then at the end I'll talk a little bit about directed evolution methods to actually engineer capsids with particular properties that are beneficial.Throughout this we've identified some of the issues that arise in this. It was initially thought that AAV vectors were non-immunogenic, but in fact there are immune responses not just to the viral payload to the therapeutic protein, but also to the viral vectors, and you're going to hear about that from Doctor Parsons. Over time, as we've come to understand these challenges, we've also been developing approaches to mitigate them. In terms of clinical trials and treatments, the very first studies were done back in the 1970s.By the early 2000, the very first clinical therapeutic was approved in China. It was actually an oncolytic virus carrying a p53 gene to treat head and neck cancers. By now there are over 40 approved treatments for various types of AAV delivered gene therapies. Of course, the ones we know a lot about are Zolgensma, which was approved in 2019, and Elevidys, which was approved last year. A number of challenges and then also a number of approaches to overcome those challenges. First of all, the preclinical data are not always sufficient to predict the response of a human patient.For example, in X-linked myotubular myopathy we had mouse and dog models that exhibited a myopathy but nothing else, and yet when we treated human patients, we discovered that patients with X-linked myotubular myopathy actually had a previously only poorly recognized hepatopathology that led to potential liver consequences following gene therapy. The animal models don't always predict the clinical outcome in humans.Also, we have small disease populations. These are rare diseases. It's important to understand the natural history of these diseases, understand the heterogeneity among the clinical population. It's very important to engage with families and with patients and communities, understand who might be at increased risk to treatment with one of these. This feeds into safety considerations. We need to think also about some of the immune responses. I think we're starting to learn, for example, with the gene therapies for Duchenne, and we know this from SMA that some patients get into trouble and others don't. We need to understand why that may be, and we don't know about the long term effects. This has been very recent.

Drs. Beggs and Parsons discuss the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at the MDA 2025 conference in Dallas, Texas, in March 2025, and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established in contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The ASPIRO clinical trial is on clinical hold since September 2021. In this part, Dr. Beggs will provide an introduction to gene-directed therapies.Alan Beggs, PhDI'm going to talk now about challenges, a little bit of background in the history and the development of AAV-mediated gene therapies, in particular for neuromuscular disorders. There are a lot of aspects about neuromuscular disease that make it a good group of conditions to target by gene replacement therapies. These are traditionally single gene disorders with known identified oftentimes protein deficiencies, so null mutations leading to lack of a protein.The primary tissue, the therapeutic target is a skeletal muscle, and so we can target that with the appropriate viral vectors. There's a major unmet medical need and substantial clinical burden for these conditions. As rare diseases, they place a very substantial burden on both health systems and patients, both economically and in terms of personal difficulties.I like to think about gene therapy, which is generically used for one category of this, to really think about gene-directed therapy. So this would be any therapy directed at the nucleic acids that are either encoding our DNA or are encoding the messenger RNA transcripts. So one approach to a gene-directed therapy can be directed at the RNA level. I think you're all familiar with the Exon-skipping approaches that target mRNA splicing.There are other methods for either knocking down toxic gain of function messenger RNAs, and there are methods now being developed to edit messenger RNAs. So this represents one class of gene therapy. You can also approach gene therapy at level of DNA by editing or changing the DNA in situ. So various CRISPR-Cas9-based approaches. There's now prime editing and other approaches for genetic engineering that target specific locations, often using bacteria endonucleasis that target with oligenucleotides that target specific sites.And then finally, there's gene replacement therapy, which is what we're going to spend most of our time on today, which really aims to not take away what's there and replace it, but to replace the missing protein product by providing a copy of the healthy or the complete wild type gene. Often, it can either be integrated into the chromosomes or remain extrachromosomal.So whether or not that happens really depends on the type of vector or approach you use. You can see here a number of different approaches for transferring in a therapeutic gene. The two most commonly used in clinical trials are lentivirus and AAV, and they have different strengths and weaknesses. Lentiviruses are used frequently for hematologic diseases.Lentivirus is a member of the retrovirus family and has the characteristic that it actually integrates into the DNA. So lentiviral treatments tend to be long-acting. However, they also suffer from the risk that by integrating into the DNA, you might have site-directed mutagenesis. And there have been known instances of cancers that arose through integration at the wrong site.In the next part, Dr. Beggs will cover the history and challenges in the development of AAV-mediated gene therapies.

Julie A. Parsons, MDHaberfield Endowed Chair in Pediatric Neuromuscular DisordersProfessor of Clinical Pediatrics and NeurologyUniversity of Colorado School of Medicine, Children's Hospital ColoradoAurora, CO, USANow, with our collective experience, we can at least put together the information that we have in terms of what can we expect and what's the timeline that we expect in terms of our patients having reactions. I will tell you, and I've said this multiple times, when I deliver a gene transfer therapy, I hold my breath for 2 months. Now, maybe it's going to have to be extended to a year, but it's typically at least for 2-3 months. It's like, okay, what's going to happen? You sit on the edge of your seat on pins and needles, going, "Is this kid going to be okay or not?" I think that's the appropriate response to have in terms of the light of things that have happened over time. We have to be really careful.We have a little bit of a framework now to say, when do we need to be really excited? We know that our patients, most all of them, are going to develop a transaminitis, and that ends up happening early on, but we get a couple of peaks. We get really excited that the 4-8 week time point with transaminitis looking for liver failure.The cholestatic liver disease that happened in the patients with X-linked MTM happened a little bit later, so Week 2, all the way out to six months afterwards. The acute cardiomyopathy a little bit earlier, so we're looking a little bit earlier for that effect. TMA, usually the end of the first week to about 2 weeks is when we would expect that to come in. Then the transgene-related myositis and immune-mediated myocarditis, weeks, maybe 2 to a couple of months.How do we adapt our gene transfer programs to the clinical trial experience? I think that there are a couple of points that are important. One is that the outline that I showed you, there are some disease-agnostic issues that come up with transaminitis, with TMA. I think there are some final common pathways related to the immune responses that we see with these patients. Then there are going to be some disease-specific disorders that are going to come up with each of these therapies and agents.We need to have good communication, honestly, in real-time. I still don't know that we have a good mechanism for that as a community, but to share these adverse events that come up so that we can all learn as a collective about what to expect, what to anticipate, and how to best take care of our patients. We know now how we need to monitor patients closely from a laboratory standpoint, from a clinical exam standpoint, and we really need to work on how are we going to mitigate some of these risk issues that we have with these patients.I think the collaborative aspect, particularly at meetings like this, is important. Last year, for the people that were at MDA, you remember that we really spent a lot of time looking at gene transfer delivery. Many of us got together as providers and actually met together to say, "Is there something that we can think about in terms of best practice or consensus in terms of how we would want to manage patients or how we'd want to share information?"Now, actually, on the MDA website, we really do have some guidelines, and there will be a publication coming out shortly that we'll have this available to everybody again. It's not necessarily the right answer, but it's at least from a collective experience, what's the best way that we can go forward? Some of the suggestions were that the adverse events right now, we can put them into some a predictable timeline, but we don't really know all the risks at the time of dosing.We know that gene transfer therapy can be safe for the right patient at the right time for the right disorder. That's really what we want to do. There's a Neurotherapeutic window between efficacy and toxicity. How are we adjusting that? What are we working on to make sure that we're getting that right? The preclinical data is helpful, but it's never the full story. Any time we go from a homogeneous population that we see in a clinical trial to a heterogeneous population, as we throw this out to the world, we're going to have new issues that arise, and we need to be aware and ready for those.We want to be able to predict what happens, but we can't always do that. Then follow-up is so important. The post-marketing study, sharing adverse events, sharing experiences, I think, is really important as well. Clinicians really should be familiar with this entire field before ever delivering gene transfer therapy. I don't think that every site should be delivering gene transfer. I think that from an institutional standpoint, you need to be ready. You need to have a team who knows what they're doing and knows how to handle the issues and the problems, or you need to have lifelines set up in advance if you're going to deliver these treatments.

Nicola Longo MD, PhD, and Mark Roberts, MDNicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfesor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discuss the current status of gene therapies in rare neuromuscular disorders in this 8-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th-7th 2025 and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses.The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Roberts will discuss immune responses and other safety concerns related to gene therapies.Mark Roberts, MDUndoubtedly, the immune system is a major issue in these patients. It would be fantastic if we could immunotolerize our patients and indeed prevent the rejection of the therapy. We've talked about the fact that these are viral vectors and of course there may be high seroprevalence of antibodies to these viral vectors, and it's very important in the pre-screening of patients who might be eligible to understand that at the beginning. These of course can have developed over the years and of course can be part of immunological memory and therefore extremely difficult and probably impractical to actually shift.On giving the treatment though as I think we're all aware there is this problem of the innate immunity and potential therefore for acute toxicities and then a learned or adaptive response with cytotoxic T cells and antibodies which may of course become high tighter neutralizing antibodies and potentially antibodies not only against the viral vector, even the functional protein, even the transgene are all theoretical possibilities with time. The capsid, the transgene, and even the protein product can all potentially induce an immunological event. Of course, all of these would lead to both potential patient changes and then a lack of efficacy of the treatment.Indeed, there have been some serious and indeed fatal problems in the gene therapy development program as I think we're all aware. Though many of these are thankfully been overcome. Spinal muscular atrophy has a gene therapy which is licensed, but there were early patients who actually had significant problems. A patient of just 6 months of age who developed kidney failure, two other patients who actually developed liver failure.In Duchenne muscular dystrophy, a very common condition, again there were significant issues and crucially in these patients who all have cardiomyopathy, it was heart failure and cardiac arrest that were big concerns and pulmonary edema and this was seen even with a CRISPR-based technology and is perhaps is best known but has been addressed the excellent myotubular myopathy patients, four patients died and crucially quite a long time after the gene therapy emphasizing the need to monitor these patients extremely carefully and these patients died of cholestatic liver failure albeit that they had a degree of liver dysfunction.That's changed our screening of course of patients, we're now all looking in myotubular patients for liver involvement and Rett syndrome as well. Now these immunoprophylaxis treatment regimes to hopefully try and reduce the immunological reaction against the gene are certainly evolving.This is just a summary of some of the other immunosuppressive regimes used in other disorders, for example, spinal muscular atrophy, but Pompe and MPS as examples of LSDs. Certainly these regimes will continue to evolve and are going to be very important in seeking to make sure that these treatments are effective. It reminds me somewhat of what's happened with enzyme replacement therapy that the use of these immunological strategies in infants has revolutionized the utility of those treatments in early patients.In the next part, Dr. Roberts will discuss lessons learned from gene therapy trials.

Nicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only. This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Longo will discuss ongoing gene therapies in lysosomal disorders.Nicola Longo MD, PhDI'm going to present to discuss some example of ongoing gene therapy for lysosomal disorder. There are gene therapy in development for both Fabry disease and some of this involve ex vivo gene therapy, many others involve systemic administration with an AAV, Gaucher disease type 1 that affect the periphery, and Gaucher disease type 2, where the replacement should occur within the central nervous system because this condition affects the brain. There is already one approved gene therapy for lysosomal disorder, which is for the early onset metachromatic leukodystrophy. This has been approved both in Europe and now even in the United States, which consists of ex vivo gene therapy with the administration of an extra gene that restore the function of the defective enzyme. Now there are many others that are ongoing for the same indication. There are gene therapy programs for GM1 and GM2 gangliosidosis, and at least one for Krabbe disease. It is important to know that some of these condition are actually included in the recommended uniform screening panel. Basically, we would have access to patients in a timely manner for some of these conditions. Then there are several gene therapy under development for the mucopolysaccharidoses, including MPS-IH, MPS-II, MPS-IIIA and MPS-IV.There are different type of lysosomal disorders, the one caused by mutation, integral membrane protein, not enzyme within the lysosome, but protein that are present on the membrane of the lysosome. This gene therapy that have been tested, it is for cystinosis, that it is caused by a defective lysosomal and for Danon disease, which is caused by a deficiency of an integral membrane part. Finally, one lysosomal disorder, which obviously seems a metabolic condition, but it is really not, is glycogen storage disease type 2 or Pompe disease, in which there is the intralysosomal accumulation of glycogen. There are several ongoing clinical trials to try to correct the problem in this condition.Now, I'm going to discuss some of the most advanced program in the lysosomal storage disorder. This include one for Fabry, which is on an accelerated approval pathway with phase 1 and 2 data, one for Gaucher disease type 1. Obviously, I'm going to discuss the one that has been already approved for metachromatic leukodystrophy. There is one for Hunter syndrome, and the difference of the one for Hunter syndrome, it is an example of the direct administration of gene therapy within the central nervous system.Finally, there is one ongoing for glycogen storage disease type 2 or Pompe disease in adult patients. In gene therapy for metachromatic leukodystrophy, it was the first gene therapy approved for lysosomal disorder in human, and this requires harvesting the CD34 cell from affected patient and then introducing the [inaudible 00:04:32] gene back in this cell, and then placing them back inside the patient again. This has been very effective in patients who were treated early, and obviously, the treatment needs to occur before there is irreversible brain damage in this patient.In the next part, Dr. Roberts and Longo will discuss treatment with gene therapies.

In this episode of the Oncology Brothers podcast, we are joined by Dr. Komal Jhaveri, a breast medical oncologist at Memorial Sloan Kettering, to discuss the evolving landscape of metastatic hormone receptor positive breast cancer, particularly focusing on low and ultra-low HER2 expression. Key topics include: • The significance of the DESTINY Breast-04 and DESTINY Breast-06 studies and their impact on treatment options. • The definition and implications of low and ultra-low HER2 expression in clinical practice. • The importance of HER2 testing and the dynamic nature of HER2 expression in tumors. • Treatment sequencing strategies, including the use of antibody-drug conjugates (ADCs) like trastuzumab deruxtecan (TDXd) and sasituzumab govitecan. • Management of treatment-related toxicities, including ILD, nausea, and alopecia. Join us for an insightful discussion that aims to keep healthcare professionals updated on the latest advancements in cancer care. Follow us on social media: •⁠  ⁠X/Twitter: https://twitter.com/oncbrothers •⁠  ⁠Instagram: https://www.instagram.com/oncbrothers •⁠  Website: https://oncbrothers.com/ Don't forget to subscribe for more episodes and insights!

Join Professor Iain McInnes for the latest episode of Discussing RA on The Immune-Mediated Inflammatory Disease Forum. In this episode, he and Doctor Reike Alten will be reviewing two papers. The first paper by Kameda et al. assessed long-term safety and efficacy of UPA over 5 years in Japanese patients with moderate-to- severe active RA and an inadequate response to stable doses of csDMARDs-IR. The second paper by Salvato et al. assessed the impact of chronic oral low-dose GCs on the efficacy and retention rates of JAK inhibitors compared to other mechanisms of action therapies in a cohort of RA patients with inadequate response to TNFi.

In this week's episode, Blood editor Dr. Laurie Sehn interviews three of the latest Blood authors: Drs. Vijay Sankaran, Ruud Delwel, Françoise Kraeber-Bodere. Two studies on the MECOM gene have been paired in this episode, analyzing new groundwork for potential novel myeloid differentiation therapies via repression of MECOM restoring enhancer mediated CEBPA expression. We'll also hear about the results of CASSIOPET, imaging companion study of the CASSIOPEIA trial, and how achieving negativity in PET before starting maintenance therapy is significant even in patients who still show residual disease in the bone marrow.Featured ArticlesCEBPA repression by MECOM blocks differentiation to drive aggressive leukemiasMECOM is a master repressor of myeloid differentiation through dose control of CEBPA in acute myeloid leukemia Prognostic value of premaintenance FDG PET/CT response in patients with newly diagnosed from the CASSIOPEIA trial

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/CC/AAPA information, and to apply for credit, please visit us at PeerView.com/ZYK865. CME/MOC/CC/AAPA credit will be available until December 15, 2026.Tackling CRSwNP Management With Emerging Biologic Therapies Targeting Epithelial Cytokines: Game On! In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/CC/AAPA information, and to apply for credit, please visit us at PeerView.com/ZYK865. CME/MOC/CC/AAPA credit will be available until December 15, 2026.Tackling CRSwNP Management With Emerging Biologic Therapies Targeting Epithelial Cytokines: Game On! In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/CC/AAPA information, and to apply for credit, please visit us at PeerView.com/ZYK865. CME/MOC/CC/AAPA credit will be available until December 15, 2026.Tackling CRSwNP Management With Emerging Biologic Therapies Targeting Epithelial Cytokines: Game On! In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/CC/AAPA information, and to apply for credit, please visit us at PeerView.com/ZYK865. CME/MOC/CC/AAPA credit will be available until December 15, 2026.Tackling CRSwNP Management With Emerging Biologic Therapies Targeting Epithelial Cytokines: Game On! In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/CC/AAPA information, and to apply for credit, please visit us at PeerView.com/ZYK865. CME/MOC/CC/AAPA credit will be available until December 15, 2026.Tackling CRSwNP Management With Emerging Biologic Therapies Targeting Epithelial Cytokines: Game On! In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca Pharmaceuticals.Disclosure information is available at the beginning of the video presentation.

Obesity affects more than 1 billion people worldwide and is recognized by the World Health Organization as a chronic, relapsing disease. WHO recently published a guideline in JAMA on the use and indications of GLP-1 therapies for the treatment of #obesity in adults. Francesca Celletti, MD, PhD, and Ezekiel Emanuel, MD, PhD, join JAMA Editor in Chief Kirsten Bibbins-Domingo, PhD, MD, MAS, to discuss. Related Content: World Health Organization Guideline on the Use and Indications of Glucagon-Like Peptide-1 Therapies for the Treatment of Obesity in Adults

Nicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established, and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses.The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas.In this part, Dr. Roberts will discuss lysosomal disorders and the potential for gene therapies.Mark Roberts, MDI'm going to give an overview of what is gene therapy, emphasizing the current challenges and the development issues and needs that there will be as we try and enable gene therapy for our patients, particularly those with lysosomal storage disorders.I'm going to try and make a case for why lysosomal storage disorders are an extremely good group of conditions for the potential benefits of gene modifying therapies. Firstly, whilst we all recognize that these conditions are inherently individually rare, they're certainly severe. Collectively, with over 70 LSD disorders, 1 in 5,000 may be afflicted by these conditions ultimately in their life and can be detected, for example, by newborn screening programs.Secondly, there's certainly a significant clinical burden with these patients with the current standard of care, so a large unmet need exists. Existing enzyme replacement therapies have undoubtedly changed the natural history of many of these conditions, but there are limitations and often initial benefits and later deteriorations.Unfortunately, for most lysosomal storage disorders, it's only symptomatic treatments and indeed, care that is available for these patients with no specific treatment. Thirdly, these conditions are extremely well-characterized, monogenic singleton and problems of inborn errors of metabolism. We know the functional protein that is deficient in these conditions. Because of that, and knowing that these are critical for lysosomal function, and using preclinical models, we can model the potential benefits of gene therapies very well in a number of systems, including, of course, soon, muscle chip experiments as well.Finally, with these conditions, they may potentially be really useful targets whilst not perhaps curing the condition, at least ameliorating the phenotype, and enabling the addition of other treatments as well, potentially. I've noted, some of these therapies can be directly delivered to certain tissues, so muscle tissue, which is my main interest, but also, crucially, the central nervous system, which is very important when we consider ameliorated phenotypes, for example, treated by enzyme replacement therapy, but where the children who become the adults have significant learning disability as a major component to their problems.In the next part, Dr. Roberts will discuss vectors, different strategies, modes of administration, and targets in gene replacement therapies.

This episode is sponsored by Zomedica. Live from dvm360's Fetch Kansas City conference, host Adam Christman, DVM, MBA, sits down with Nicole Westfall, MBA, senior vice president of marketing at Zomedica. In this episode, they explore the benefits of PulseVet Shock Wave Therapy and The Assisi Loop, highlighting how these innovative tools provide noninvasive, effective pain management for patients suffering from chronic conditions.

In this episode of the Heart podcast, Digital Media Editor, Professor James Rudd, is joined by Dr John Sapp from Halifax, Nova Scotia, Canada. They discuss his review paper all about ventricular tachycardia in terms of drug and device therapies, plus advances in VT ablation. If you enjoy the show, please leave us a positive review wherever you get your podcasts. It helps us reach more people—thanks! Link to published paper: https://heart.bmj.com/content/early/2025/10/07/heartjnl-2024-325370  

Autologous cell therapies have shown the promise of single-dose, curative treatments for patients with advanced cancers. But even with major scientific and regulatory progress, the field now faces its toughest challenge yet: achieving sustainable, scalable manufacturing for highly personalized therapies. In this episode of Off Script, we spoke with Jason Bock, co-founder and CEO of CTMC, a purpose-driven cell therapy accelerator. Jason discusses why scale remains the central barrier for autologous therapies, the significance of CTMC's partnership with MD Anderson Cancer Center, and how fit-for-purpose manufacturing models can shorten vein-to-vein time and bring transformative treatments to patients faster.

Master cardiovascular disease prevention! Learn how to apply the new PREVENT risk calculator, use the CPR framework for risk reclassification, and interpret ApoB and Lp(a) in modern lipid management. We're joined by Dr. Laurence Sperling to break down what's new in ASCVD risk assessment and prevention. Claim CME for this episode at curbsiders.vcuhealth.org! Patreon | Episodes | Subscribe | Spotify | YouTube | Newsletter | Contact | Swag! | CME Show Segments Intro Case 1 from Kashlak  Risk assessment framework ApoB and Non-HDL cholesterol Lipoprotein(a) and its importance The PREVENT risk score and its implications Statins and other lipid lowering therapies The role of diet and lifestyle in lipid management Case 2 from Kashlak Familial Hypercholesterolemia diagnosis and management Key takeaways Outro Credits Producer, Writer, Shownotes, Infographic, and Cover art: Ben Furman, MPH  Hosts: Matthew Watto MD, FACP; Paul Williams MD, FACP    Reviewer: Leah Witt, MD Showrunners: Matthew Watto MD, FACP; Paul Williams MD, FACP Technical Production: PodPaste Guest: Laurence Sperling, MD  Disclosures Dr. Sperling reports no relevant financial disclosures. The Curbsiders report no relevant financial disclosures. Sponsor: Bucksbaum-Siegler Institute If you want to learn more about what the Bucksbaum-Siegler Institute is doing and to nominate someone for the Clinical Excellence Award—you can check them out today. Visit bucksbauminstitute.uchicago.edu. Sponsor: Grammarly  Sign up for FREE and experience how Grammarly can elevate your professional writing from start to finish. Visit Grammarly.com/podcast.  Sponsor: Continuing Education Company Use promo code Curb30 to get 30% off all online courses and webcast. Visit CMEmeeting.org/curbsiders to learn more. Sponsor: Freed Use code: CURB50 to get $50 off your first month when you subscribe!

Please visit answersincme.com/860/99120473-replay to participate, download slides and supporting materials, complete the post test, and get a certificate. In this activity, experts in oncology discuss the latest advances in HER2- and TROP2-directed ADCs for the management of advanced NSCLC, and how these approved and emerging ADCs may impact patients' treatment algorithms. Upon completion of this activity, participants should be better able to: Identify the rationale for targeting HER2 and TROP2 in the treatment of non-small cell lung cancer (NSCLC; Discuss the clinical impact of approved and emerging HER2- and TROP2-directed antibody-drug conjugates (ADCs) in NSCLC; and Formulate evidence-based strategies for the individualized management of patients with NSCLC using HER2- and TROP2-directed ADCs.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into the exciting, yet challenging, landscape of gene therapies and their potential to revolutionize healthcare. Recent scientific advancements have pushed the boundaries of what's possible, offering potential lifetime cures for diseases once considered incurable. However, this breakthrough comes with a significant economic caveat: the staggering cost of these therapies, often ranging between $3 to $4 million per patient. This price tag presents a formidable challenge to current healthcare infrastructures.The disconnect between these innovative treatments and existing payment systems is evident in what industry experts term the "$4 million payment problem." Therapies like Lenmeldy and Hemgenix highlight this issue. Lenmeldy, for instance, can prevent metachromatic leukodystrophy with a single infusion priced at $4.25 million, while Hemgenix offers a cure for hemophilia B at $3.5 million. These therapies effectively convert lifelong treatment costs into a singular, substantial payment, challenging traditional insurance models that are built to spread costs over time.The primary obstacle is not the efficacy of these treatments but rather the financial and logistical infrastructures needed to support them. The current insurance model is ill-equipped to handle such large, one-time payments. Employers who often provide health insurance face a dilemma: investing millions in curing an employee who might leave the company shortly after receiving treatment could result in significant financial risk and disincentivizes employers from covering such therapies.Enter Aradigm Health, which has emerged as a potential solution to this conundrum. Aradigm aims to create an "infrastructure layer" specifically for these high-cost cures. With $20 million in funding backing their initiative, Aradigm seeks to pool financial risk across multiple employers, thus mitigating the impact of substantial individual claims. Their model involves employers contributing a fixed monthly fee into a shared fund that covers these expensive treatments when needed. This approach distributes financial volatility across a broader base rather than placing it on individual employers.Aradigm's strategy is not only about financial solutions but also about streamlining logistical complexities associated with delivering gene therapies. Their patient journey management includes coordinating with biotech companies for manufacturing schedules, arranging travel and accommodation for patients and families, and ensuring seamless insurance paperwork handling. This comprehensive support system reduces barriers that often delay or disrupt treatment delivery.Operating as a public benefit corporation with a "cost-plus" model, Aradigm ensures that any surplus from lower-than-expected claims is returned to employers rather than kept as profit. This aligns incentives towards patient care rather than profit maximization. Their approach highlights a critical need within the biotech and pharmaceutical industries: developing adaptable infrastructures that align with rapid scientific advancements.Meanwhile, Amgen has secured significant ground in 2023 with its second FDA approval for Uplizna in treating generalized myasthenia gravis—a chronic autoimmune neuromuscular disorder characterized by varying degrees of skeletal muscle weakness. Uplizna's mechanism involves targeting CD19 on B cells implicated in autoimmune diseases' pathogenesis. This expansion marks an advancement in therapeutic options for patients and underscores Amgen's growing footprint in treating complex autoimmune conditions.GlaxoSmithKline has also made headlines with Blujepa, marking it as the first new class of antibiotics for gonorrhea in over three decades while receiving approval for treating uncomplicSupport the show

Discover how non-medical therapies like cognitive behavioral therapy, mindfulness, exercise, and creative outlets provide powerful alternatives to medication for treating depression and anxiety. Learn which holistic approaches are backed by science and how to start your recovery today. Read more at https://missionconnectionhealthcare.com/our-facilities/washington/outpatient-mental-health-services-seattle/ Mission Connection City: San Juan Capistrano Address: 30310 Rancho Viejo Rd. Website: https://missionconnectionhealthcare.com/

In this episode, I sit down with neurological rehab specialist Dr. Nick Schmidlkofer to break down why stroke recovery doesn't stop after one or two years, despite what many patients are told. We discuss why some people recover 90% while others only improve halfway, and how factors like inflammation, diet, targeted at-home exercises, HBOT therapy, red light therapy and other combinations of therapies influence outcomes.Dr. Nick also explains how his clinic combines vestibular rehab, vagus and peripheral nerve stimulation, red-light therapy, and hyperbaric oxygen to re-activate damaged brain regions and accelerate neuroplasticity. If you or a loved one is still struggling years after a stroke, this conversation shows why recovery is still possible.

Send us a textHow ketosis and ketogenic diets work and how these tools can improve metabolic health, brain function, and even cancer management.Topics Discussed:Organs have different fuel preferences: brain strongly prefers glucose, heart prefers fatty acids, skeletal muscle is flexible and likes fat/ketones.Humans evolved with high metabolic flexibility; regular ketosis was normal for ancestors, but today most people never experience it.“Keto flu” is largely glucose withdrawal plus electrolyte/sodium loss; proper salt and hydration prevent most symptoms.Classic medical ketogenic diet is ~90% fat (historically saturated); modern versions often use more monounsaturated fats, MCTs, and higher protein.Saturated fat is not inherently atherogenic in the context of weight stability or caloric deficit; excess calories from any source can dysregulate metabolism.Exogenous ketones (e.g. BHB) provide energy, reduce ROS, stabilize membranes, increase inhibitory tone (GABA), and have hormone-like signaling effects independent of diet.Cancer cells often show Warburg effect (damaged mitochondrial respiration → heavy reliance on glycolysis); lowering glucose and raising ketones can stress cancer cells.True keto-adaptation for athletic performance requires 6–12 weeks; after that, elite athletes can match or exceed prior high-carb performance at sub-maximal and endurance efforts.Practical Takeaways:Therapeutic carbohydrate restriction (50–100 g/day for many people) plus occasional fasting or ketone supplements can restore metabolic flexibility with far fewer side effects than strict keto.Prioritize whole-food fats (eggs, fatty fish, beef, olive oil, butter/lard) and minimize processed keto products loaded with seed oils.Supplementing BHB (salts or esters) or MCT oil can ease the transition into ketosis, boost ketones without strict dieting, and may support brain and metabolic health.Regularly check basic blood markers (glucose, lipids, electrolytes) and consider an OmegaQuant test; optimizing metabolic health is one of the strongest preventable steps against cancer, neurodegeneration, and heart disease.Supplemental Ketone (BHB):KetoCitra—Ketone body BHB with potassium, calcium & magnesium, formulated with kidney health in mind. Use code MIND20 for 20% off.*Not medical advice.Support the showAffiliates: Lumen device to optimize your metabolism for weight loss or athletic performance. MINDMATTER gets you 15% off. AquaTru: Water filtration devices that remove microplastics, metals, bacteria, and more from your drinking water. Through link, $100 off AquaTru Carafe, Classic & Under Sink Units; $300 off Freestanding models. Seed Oil Scout: Find restaurants with seed oil-free options, scan food products to see what they're hiding, with this easy-to-use mobile app. KetoCitra—Ketone body BHB + electrolytes formulated for kidney health. Use code MIND20 for 20% off any subscription (cancel anytime) For all the ways you can support my efforts

Discover nurse-led strategies to support patients with adherence to oral therapies for the management of chronic lymphocytic leukemia and mantle cell lymphoma Credit available for this activity expires: 12/05/2026 Earn Credit / Learning Objectives & Disclosures: https://www.medscape.org/viewarticle/mastering-oral-therapies-cll-and-mcl-essential-strategies-2025a1000xko?ecd=bdc_podcast_libsyn_mscpedu

Thanks for listening to The Peptide Podcast.  Today, we're diving into GLP‑1 therapies, including dual GIP/GLP‑1 agonists and the emerging triple agonists, and talking about how these peptides are typically dosed, what people are experimenting with, and what the research — or real-world experience — tells us so far. Read the Full Episode Transcript: https://pepties.com/glp-1-therapies-strategies-weekly-micro-dose-and-maintenance/ Dr. Nikki's Qualifications: https://bifat.life/about/ Related Links/Products Mentioned: Peptide Podcast Partners Page https://pepties.com/partners/ BioLongevity Labs (Purchase Peptides online) Use our link and enter COUPON CODE: PEPTIDEPODCAST at checkout to receive 15% off your total order https://go.biolongevitylabs.com/aff_c?offer_id=1&aff_id=1582&aff_sub=PEPTIDEPODCAST Momentous Supplements (we use Creatine, Vital Aminos, Whey Protein) https://crrnt.app/MOME/OqGQOxGA LMNT – More Salt, Not Less.  https://elementallabs.refr.cc/default/u/johnjavit Thorne Supplements (we use Omega-3 with CoQ10, Red Yeast Rice, Zinc) https://get.aspr.app/SH1KvW Organifi Creatine and Shilajit Gummies http://rwrd.io/rlbkajm?c MitoZen (methylene blue for Cognitive Function, Anti-Aging, Mental Clarity) https://www.mitozen.com/ref/cnlwiztypt/ For skin and hair health (Copper Tripeptide-1)  Visit Luminose by Entera for an exclusive offer for Peptide Podcast listeners!  ** Promo code PEPTIDEPODCAST at checkout for 10% off an order or 10% off the first month of a subscribe-and-save. ** https://www.enteraskincare.com/?rfsn=8906839.f93c72

Jesse Levey, CEO and Founder of Longevity Health, has a focus on extending the health span not just the lifespan of more people by providing access to tools and preventative health information. Lessons learned from expensive concierge medical services are being applied to a broader population, driving down health costs and democratizing longevity medicine. Using AI to build a scalable and personalized approach to wellness not just sick care, Longevity Health aims to make an AI doctor available around the globe.  Jesse explains, "Our mission is to help a billion people live to a hundred in good health. That's the vision. And the way that we get there is by building an AI doctor trains of ed on longevity medicine and distributed around the world. We have a three-phased business model. Phase one is to take the sort of hundred-thousand-dollar-a-year longevity concierge experience and deliver it for $10,000. So we've taken this really high-end experience that combines an executive physical with a longevity concierge physician and a team to help you implement the recommendations. And we've delivered that for $10,000. That's been around for about two years."  "Phase two takes that down to $1,000 with the help of AI. So it reduces the time spent by humans and the need to spend a lot of time with clinicians. And so it's a mix between clinician time and AI interactions. And then eventually, as the regulation allows and as the technology improves, we believe that we'll be able to deliver this experience for as low as $10 a month or $100 a year via an AI doctor. And so that's the future. That's what we're building towards." #LongevityHealth #Longevity #HealthyAging #HealthSpan #FunctionalMedicine #HealthAI #Aging longevityhealth.me Download the transcript here

Jesse Levey, CEO and Founder of Longevity Health, has a focus on extending the health span not just the lifespan of more people by providing access to tools and preventative health information. Lessons learned from expensive concierge medical services are being applied to a broader population, driving down health costs and democratizing longevity medicine. Using AI to build a scalable and personalized approach to wellness not just sick care, Longevity Health aims to make an AI doctor available around the globe.  Jesse explains, "Our mission is to help a billion people live to a hundred in good health. That's the vision. And the way that we get there is by building an AI doctor trains of ed on longevity medicine and distributed around the world. We have a three-phased business model. Phase one is to take the sort of hundred-thousand-dollar-a-year longevity concierge experience and deliver it for $10,000. So we've taken this really high-end experience that combines an executive physical with a longevity concierge physician and a team to help you implement the recommendations. And we've delivered that for $10,000. That's been around for about two years."  "Phase two takes that down to $1,000 with the help of AI. So it reduces the time spent by humans and the need to spend a lot of time with clinicians. And so it's a mix between clinician time and AI interactions. And then eventually, as the regulation allows and as the technology improves, we believe that we'll be able to deliver this experience for as low as $10 a month or $100 a year via an AI doctor. And so that's the future. That's what we're building towards." #LongevityHealth #Longevity #HealthyAging #HealthSpan #FunctionalMedicine #HealthAI #Aging longevityhealth.me  Listen to the podcast here

Silviu Itescu, CEO of Mesoblast (MESO), discusses their stem cell work and their FDA-approved products. Their latest work is to treat acute graft vs host disease in children, and he says they've seen “terrific launch results.” He talks about gaining Medicaid coverage and adoption by private insurance plans. Silviu covers their future drug pipeline and how they are targeting heart disease and inflammation.======== Schwab Network ========Empowering every investor and trader, every market day.Options involve risks and are not suitable for all investors. Before trading, read the Options Disclosure Document. http://bit.ly/2v9tH6DSubscribe to the Market Minute newsletter - https://schwabnetwork.com/subscribeDownload the iOS app - https://apps.apple.com/us/app/schwab-network/id1460719185Download the Amazon Fire Tv App - https://www.amazon.com/TD-Ameritrade-Network/dp/B07KRD76C7Watch on Sling - https://watch.sling.com/1/asset/191928615bd8d47686f94682aefaa007/watchWatch on Vizio - https://www.vizio.com/en/watchfreeplus-exploreWatch on DistroTV - https://www.distro.tv/live/schwab-network/Follow us on X – https://twitter.com/schwabnetworkFollow us on Facebook – https://www.facebook.com/schwabnetworkFollow us on LinkedIn - https://www.linkedin.com/company/schwab-network/About Schwab Network - https://schwabnetwork.com/about

In Part 2 of the 2026 pipeline series, host Carolyn Liptak welcomes Dr. Amanda Frick, Senior Clinical Manager, Strategic Clinical Intelligence at Vizient, to explore the advanced therapies pipeline: cell therapies, gene therapies, tissue-engineered products, and combination advanced therapy products. The discussion explores major pipeline trends, six leading products to watch, and the growing innovation expected to shape clinical practice in 2026.   Guest speakers:  Amanda Frick, PharmD, BCPS Senior Clinical Manager, Strategic Clinical Intelligence Spend Management Vizient Host: Carolyn Liptak, MBA, BS Pharm Pharmacy Executive Director Center for Pharmacy Practice Excellence (CPPE) Vizient    Show Notes: [00:05] — Introduction Announcer opens the episode. Host Carolyn Liptak introduces the focus on advanced therapies: cell & gene therapies, tissue-engineered products, and combination products. Guest: Dr. Amanda Frick, Senior Clinical Manager, Strategic Clinical Intelligence at Vizient. [01:07] — Defining Advanced Therapies FDA groups cell and gene therapies within advanced therapies. Total FDA-approved advanced therapies: 46. Amanda monitors 29 drug-like therapies within that group. [02:01] — Pipeline Size and Approval Activity S. pipeline: 264 agents in development. About 10 agents approach FDA decision annually. Actual approvals: 5–7 per year on average. [02:56] — Big-Picture Trends in Cell & Gene Therapy Oncology dominates 40–50% of all CGTs in development. Expanding into autoimmune, neurology, and earlier-phase therapies for diabetes, angina, osteoarthritis. Movement toward allogeneic ("off-the-shelf") therapies Designed to overcome limits of autologous cell manufacturing. Reduces wait time and manufacturing failures. Resurgence of therapeutic vaccines Currently 3 approved (Sipuleucel-T, Talimogene, Papzimeos ). 20+ vaccines in the pipeline, largely targeted to cancer. CE program coming Jan 29. [06:13] — Therapy #1:  Tabelecleucel or Tab-cel (Allogeneic EBV-Specific T-Cell Therapy) First allogeneic T-cell therapy expected in the U.S. For EBV-positive post-transplant lymphoproliferative disorder (PTLD). “Off-the-shelf” and donor-derived. [07:07] — Clinical Need & Outcomes Currentstandard of care: rituximab. After relapse, survival

Eye On Research | Session 2: Adjuvant Therapies – What's New Today? (And What Can You Do About It!?) Join us for Session 2 of Eye On Research, presented by A Cure In Sight

How should retina specialists integrate next-generation anti-VEGFs into everyday wet AMD care? In this episode of “Anti-VEGFs: The Next Generation,” David Miller, MD, speaks with Esther Kim, MD, and Ehsan Rahimy, MD, about real-world issues facing wet AMD care, such as navigating crowded drug fridges, attempting interval extensions, and switching from legacy agents to next-generations treatments.  This editorially independent series is supported with advertising by Regeneron.

How should retina specialists integrate next-generation anti-VEGF agents into real-world DME care? In this episode of “Anti-VEGFs: The Next Generation,” David Miller, MD, speaks with Esther Kim, MD, and Ehsan Rahimy, MD, about first-line agent selection, when to transition to newer options, how to incorporate steroids thoughtfully, and crafting dosing strategies that balance durability, efficacy, and adherence for working-age patients—without overpromising outcomes.This editorially independent series is supported with advertising by Regeneron.

View the Show Notes Page for This Episode Become a Member to Receive Exclusive Content Sign Up to Receive Peter's Weekly Newsletter Dom D'Agostino is a neuroscientist and professor at the forefront of metabolic therapies, including ketogenic diets, exogenous ketones, and hyperbaric oxygen. In this episode, Dom breaks down nutritional versus supplemental ketosis, defines meaningful ketone thresholds, and outlines practical ways to achieve ketosis. He explains how a ketogenic diet can support metabolic health and weight loss, and advises on how to maintain adequate protein and avoid common mistakes. Dom surveys the growing landscape of exogenous ketones—from salts and esters to 1,3-butanediol—and effective pairings like caffeine, MCT oil, and alpha-GPC. He highlights the role of ketogenic therapy in cancer (particularly glioblastoma) and its promise for neurodegenerative diseases. The conversation also covers recommended hyperbaric oxygen protocols for brain injuries and cognitive function, situations where fasting or ketones offer cognitive and anti-inflammatory benefits, and touches on the carnivore diet as a ketogenic variant with potential relevance for autoimmune and metabolic conditions. We discuss: Dom and Peter's shared interest in ketosis, and Dom's scientific journey [2:30]; Dom's work for the Navy on oxygen toxicity [7:00]; Nutritional ketosis defined: physiology, biomarkers, and how fasting and diet generate therapeutic ketones [15:00]; The historical roots of ketogenic diets in epilepsy treatment, and evidence showing ketones reduce seizure activity and strengthen brain resilience [19:00]; Dom's personal experience on the ketogenic diet: tracking macros, getting enough protein, and monitoring ketone levels [24:15]; Using a ketogenic diet for weight loss: Dom's guidance on protein, fiber, calorie tracking, lipid monitoring, and more [31:00]; Protein on ketogenic diets: Dom's rationale for higher intake and muscle preservation [38:00]; Incorporating carbohydrates into keto: timing, high-fiber foods, and other considerations [41:30]; The carnivore diet: whether this diet induces ketosis, how it functions metabolically, and why it may help individuals with autoimmune conditions [44:15]; Early exogenous ketones: how 1,3-butanediol works, its liver toxicity risk, and why ketone esters replaced it [48:15]; The progression of exogenous ketones: why BHB monoesters and ketone salts emerged as better alternatives to 1,3-butanediol for ketone supplementation [59:30]; Ketone salts: easing the transition into ketosis, dosing, and how they compare to ketone esters [1:04:00]; The differences between D- and L-β-hydroxybutyrate, and how racemic mixtures may elevate ketones longer and offer unique biological effects [1:09:30]; How ketosis may boost NAD, and why NAD supplements have fallen short so far [1:16:30]; Emerging evidence for using a ketogenic diet to treat anorexia and other psychiatric disorders [1:20:30]; Potential cognitive and performance benefits of ketone supplementation, and why pushing ketones too high can be dangerous [1:23:45]; Applications for ketone esters, and why ketone salts or MCT-blended formulations may be safer and more practical for most people [1:29:15]; The role of a ketogenic diet in treating cancer [1:34:45]; The potential of a ketogenic diet for treating Alzheimer's disease [1:45:45]; Tools for cognitive enhancement: ketones, alpha-GPC, MCT, caffeine, strategic fasting, and more [1:53:45]; Hyperbaric oxygen therapy for concussion, TBI, PTSD, and cognitive function, including protocols and dosing approaches [1:55:30]; Peter's takeaways, recommended products, and additional resources to learn more [2:03:00]; and More. Connect With Peter on Twitter, Instagram, Facebook and YouTube

How do leading oncologists interpret the abundance of molecular tests, genomic data, and biomarkers to create a lung cancer patient's treatment plan? In this episode of the 2025 NSCLC Creator Weekend™ series, our tumor board discusses the complexities of lung cancer treatment, including new systemic therapies, lung cancer staging, and the role of molecular diagnostics and liquid biopsies. --- This podcast is supported by an educational grant from Johnson & Johnson and Varian. --- SYNPOSIS The panel, featuring specialists from various institutions, discusses the specifics of sequencing therapies, the impact of targeted and immunotherapies, and the nuances of treating different patient profiles, including non-smokers and those with specific genetic mutations. The conversation also touches on the integration of new staging systems, the benefits of multidisciplinary clinics, and the ongoing evolution of cancer treatment trials. The discussion aims to provide clarity on the latest advancements and future directions in managing lung cancer, emphasizing the importance of tailored treatment plans and the potential of emerging technologies. --- TIMESTAMPS 00:00 - Introduction05:16 - Molecular Diagnostics and Liquid Biopsy21:43 - Targeted Therapy Options27:29 - Managing Toxicities and Treatment Strategies33:13 - Challenges with Immunotherapy in Special Cases34:07 - Lung Transplantation in Cancer Patients48:38 - Multidisciplinary Clinics and Collaboration01:06:29 - Future Directions --- RESOURCES ADAURA Trialhttps://www.nejm.org/doi/full/10.1056/NEJMoa2027071 Gomez NSCLChttps://pmc.ncbi.nlm.nih.gov/articles/PMC5143183/

Send us a textIn this episode, Dr. Andrew Beverstock discusses his research on urinary sodium and its relationship with growth in preterm neonates. He shares insights into the importance of sodium for neonatal growth, the methodology of his study, and the unexpected results that challenge existing literature. The conversation also touches on his diverse medical training, mentorship experiences, and his involvement in medical education and point-of-care ultrasound (POCUS). Dr. Beverstock emphasizes the significance of careful population selection in research and outlines his future research directions. Support the showAs always, feel free to send us questions, comments, or suggestions to our email: nicupodcast@gmail.com. You can also contact the show through Instagram or Twitter, @nicupodcast. Or contact Ben and Daphna directly via their Twitter profiles: @drnicu and @doctordaphnamd. The papers discussed in today's episode are listed and timestamped on the webpage linked below. Enjoy!

Dr. Ian Winship is an Associate Professor and a former Alberta Innovates Health Solutions Scholar in the Department of Psychiatry at the University of Alberta. He is also Director of the Neurochemical Research Unit there. Ian is interested in understanding how we can reduce the damage early after a stroke and ways we can improve recovery in people who had a stroke a long time ago. His research also examines changes in the brain that lead to symptoms in other brain disorders like schizophrenia. Much of Ian's free time is spent on or near the ice rink. He coaches his son's hockey team and his daughter's ringette team, as well as playing on his own recreational hockey team. In the summer, Ian enjoys being outside, traveling, visiting the mountains, and relaxing at the beach. Ian received his bachelor's and doctoral degrees in Psychology from the University of Alberta. Afterwards, he conducted postdoctoral research at the University of British Columbia before returning to the University of Alberta to join the faculty. In this interview Ian shares more about his life and science.