Podcasts about Duchenne

Dr. Neil Toupin, a child neurologist at UK HealthCare, shares insight for patients and community members about Duchenne muscular dystrophy, a rare disease, and how treatment is evolving.  Learn more about David Toupin, MD 

This episode of GEN's Touching Base spotlights the unexpected relationship between breathing low oxygen and restoration of neuron function in a mouse model of Parkinson's disease. In addition, a single gene therapy injected at birth now offers lasting protection from HIV in infant rhesus macaques. Rounding out our episode, we discuss key factors shaping this year's A-List of Top 10 U.S. Biopharma Clusters and recap the regulatory conflict surrounding Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) gene therapy, Elevidys®—including the departure of Vinay Prasad, MD, the FDA official behind the agency's brief get-tough approach.Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, PhD, and Uduak Thomas for a discussion of the latest biotech and biopharma news. Listed below are links to the GEN stories referenced in this episode of Touching Base:GLP-1 Drug Semaglutide Linked to Reduced Muscle Strength in MiceGEN, August 6, 2025Out of Thin Air: Hypoxia Reverses Parkinson's Symptoms in MiceBy Fay Lin, PhD GEN, August 6, 2025Early Gene Therapy Provides Lasting HIV Protection in Newborn PrimatesGEN, July 30, 2025StockWatch: As Prasad Exits FDA, Analysts See Benefit for Sarepta, CGT StocksBy Alex Philippidis, GEN Edge, August 3, 2025Sarepta to Resume Shipping DMD Gene Therapy to Ambulant PatientsBy Alex Philippidis, GEN Edge, July 28, 2025Sarepta Under Scrutiny, AstraZeneca's Big Bet, and AI vs. IDRsBy Alex Philippidis, Uduak Thomas, Fay Lin, PhD, Corinna Singleman, PhD, GEN, July 25, 2025About Face: Sarepta to Pause Elevidys Shipments TemporarilyBy Alex Philippidis, GEN Edge, July 21, 2025Touching Base PodcastHosted by Corinna Singleman, PhDBehind the BreakthroughsHosted by Jonathan D. Grinstein, PhDThe State of Biotech Summit Registration Hosted on Acast. See acast.com/privacy for more information.

In this episode of the Brainstorm, Sam, Nick, and Multiomics Research Analyst, Shea Wihlborg, dive into the complexities of the FDA's recent decisions and their implications for gene therapy. They discuss the controversial halt of a Duchenne muscular dystrophy treatment, the role of AI in drug discovery, and the exciting future of genomics. If you know ARK, then you probably know about our long-term research projections, like estimating where we will be 5-10 years from now! But just because we are long-term investors, doesn't mean we don't have strong views and opinions on breaking news. In fact, we discuss and debate this every day. So now we're sharing some of these internal discussions with you in our new video series, “The Brainstorm”, a co-production from ARK and Public.com. Tune in every week as we react to the latest in innovation. Here and there we'll be joined by special guests, but ultimately this is our chance to join the conversation and share ARK's quick takes on what's going on in tech today.Key Points From This Episode:Discussion on the FDA's recent halt of a Duchenne muscular dystrophy treatment due to safety concerns.Exploration of the role of AI in improving drug discovery and clinical trials.Insights into the future of gene therapies for both rare and common diseases.The potential impact of regulatory changes on innovation in the biotech industry.For more updates on Public.com:Website: https://public.com/YouTube: @publicinvestX: https://twitter.com/public

La Cámara de Diputados comenzó a tratar un proyecto de ley del legislador Bruno Sarubi que instituye el 7 de septiembre de cada año como "Día Provincial de Concientización de la Enfermedad de Distrofia Muscular de Duchenne y de Becker". Por ese motivo, Radio Diputados entrevistó a María Mendoza, mamá de Nachito Vivas, un niño con distrofia de Duchenne.

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy Elevidys and growing scrutiny over the company’s transparency following multiple patient deaths. In this week’s episode of "The Top Line," we explore Sarepta’s brief standoff with the FDA over Elevidys, a short-lived marketing pause, and the regulator’s surprise reversal on the treatment. Fierce Pharma’s Fraiser Kansteiner and Angus Liu sit down with Fierce Biotech’s Gabrielle Masson to recap the story and discuss the broader implications the Elevidys saga may have for gene therapy, the FDA and the pharmaceutical industry. To learn more about the topics in this episode: Sarepta pivots to siRNA and lays off 500 staffers as Elevidys gets box warning Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy Sarepta, bowing to FDA pressure, pauses shipments of Duchenne gene therapy Elevidys FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients Analysts demand transparency after Sarepta's roundabout disclosure of 3rd patient death Roche won't throw in the towel after DMD gene therapy Elevidys' rebuff in Europe See omnystudio.com/listener for privacy information.

Howie and Harlan discuss Dr. Vinay Prasad's departure from the FDA after a backlash against his decisions on Sarepta's Duchenne muscular dystrophy gene therapy. Also examined: AI in clinical settings, changes in NIH grantmaking, and the 60th anniversary of Medicare and Medicaid. Links: Sarepta and the FDA “STAT breaks down the confusing, heartbreaking Sarepta saga” “Sarepta Therapeutics' Duchenne therapy faces ‘arduous' path back to market, senior FDA official says” “For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak” “FDA launches probe into new Elevidys death as Sarepta, Roche stress gene therapy not at fault” “FDA Recommends Removal of Voluntary Hold for Elevidys for Ambulatory Patients” “FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients” “Controversial FDA official Dr. Vinay Prasad departs agency” “Top F.D.A. Vaccine Official Resigns, Citing Kennedy's ‘Misinformation and Lies'” “Vinay Prasad Is a Bernie Sanders Acolyte in MAHA Drag” “Timeline: The Rise and Fall of Vioxx” “What have we learnt from Vioxx?” THC in pizza dough “C.D.C. Ties 85 Cases of THC-Related Symptoms to Wisconsin Restaurant” “Tetrahydrocannabinol Intoxication from Food at a Restaurant — Wisconsin, October 2024” NIH funding “NIH is shrinking the number of research projects it funds due to a new Trump policy” NIH: Supporting Fairness and Originality in NIH Research Applications “Fearful of AI-generated grant proposals, NIH limits scientists to six applications per year” “Trump budget draft proposes NIH consolidation and 40% spending cut” Raw milk “Outbreak of Salmonella Typhimurium Infections Linked to Commercially Distributed Raw Milk — California and Four Other States, September 2023–March 2024” “The Dangers of Raw Milk: Unpasteurized Milk Can Pose a Serious Health Risk” AI in healthcare update “Ambience Healthcare Announces $243 Million Series C to Scale its AI Platform for Health Systems” “As ambient scribes face off, Doximity lures doctors with a free option” Aidoc: “AI Empowering Radiologists” “Clinical Implementation of a Combined Artificial Intelligence and Natural Language Processing Quality Assurance Program for Pulmonary Nodule Detection in the Emergency Department Setting” “Large Language Models as an Inexpensive and Effective Extra Set of Eyes in Radiology Reporting” “Current Trends in Remote and Flexible Work Options in Radiology and Perception of Impact on Radiologist Well-being” Medicare and Medicaid 60th anniversary CMS' program history “Medicare and Medicaid turn 60 – and face historic cuts decades in the making” UnitedHealth and Medicare Advantage “UnitedHealth says 2025 earnings will be worse than expected as high medical costs dog insurers” “UnitedHealthcare to exit certain Medicare Advantage markets as costs balloon, impacting 600K enrollees” “Novo Nordisk slides further on US competition, new CEO concerns” “Dems Must Really Fix Medicaid, Not Just Undo Trump's Damage to It”   Learn more about the MBA for Executives program at Yale SOM. Email Howie and Harlan comments or questions.

Howie and Harlan discuss Dr. Vinay Prasad's departure from the FDA after a backlash against his decisions on Sarepta's Duchenne muscular dystrophy gene therapy. Also examined: AI in clinical settings, changes in NIH grantmaking, and the 60th anniversary of Medicare and Medicaid. Links: Sarepta and the FDA “STAT breaks down the confusing, heartbreaking Sarepta saga” “Sarepta Therapeutics' Duchenne therapy faces ‘arduous' path back to market, senior FDA official says” “For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak” “FDA launches probe into new Elevidys death as Sarepta, Roche stress gene therapy not at fault” “FDA Recommends Removal of Voluntary Hold for Elevidys for Ambulatory Patients” “FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients” “Controversial FDA official Dr. Vinay Prasad departs agency” “Top F.D.A. Vaccine Official Resigns, Citing Kennedy's ‘Misinformation and Lies'” “Vinay Prasad Is a Bernie Sanders Acolyte in MAHA Drag” “Timeline: The Rise and Fall of Vioxx” “What have we learnt from Vioxx?” THC in pizza dough “C.D.C. Ties 85 Cases of THC-Related Symptoms to Wisconsin Restaurant” “Tetrahydrocannabinol Intoxication from Food at a Restaurant — Wisconsin, October 2024” NIH funding “NIH is shrinking the number of research projects it funds due to a new Trump policy” NIH: Supporting Fairness and Originality in NIH Research Applications “Fearful of AI-generated grant proposals, NIH limits scientists to six applications per year” “Trump budget draft proposes NIH consolidation and 40% spending cut” Raw milk “Outbreak of Salmonella Typhimurium Infections Linked to Commercially Distributed Raw Milk — California and Four Other States, September 2023–March 2024” “The Dangers of Raw Milk: Unpasteurized Milk Can Pose a Serious Health Risk” AI in healthcare update “Ambience Healthcare Announces $243 Million Series C to Scale its AI Platform for Health Systems” “As ambient scribes face off, Doximity lures doctors with a free option” Aidoc: “AI Empowering Radiologists” “Clinical Implementation of a Combined Artificial Intelligence and Natural Language Processing Quality Assurance Program for Pulmonary Nodule Detection in the Emergency Department Setting” “Large Language Models as an Inexpensive and Effective Extra Set of Eyes in Radiology Reporting” “Current Trends in Remote and Flexible Work Options in Radiology and Perception of Impact on Radiologist Well-being” Medicare and Medicaid 60th anniversary CMS' program history “Medicare and Medicaid turn 60 – and face historic cuts decades in the making” UnitedHealth and Medicare Advantage “UnitedHealth says 2025 earnings will be worse than expected as high medical costs dog insurers” “UnitedHealthcare to exit certain Medicare Advantage markets as costs balloon, impacting 600K enrollees” “Novo Nordisk slides further on US competition, new CEO concerns” “Dems Must Really Fix Medicaid, Not Just Undo Trump's Damage to It”   Learn more about the MBA for Executives program at Yale SOM. Email Howie and Harlan comments or questions.

A top official at the Food and Drug Administration steps down after the agency changes course on a treatment for Duchenne muscular dystrophy. What does this episode say about the Trump Administration's approach toward drug development and innovation? Plus, Robert F. Kennedy Jr. says he plans to overhaul the National Vaccine Injury Compensation Program. Learn more about your ad choices. Visit megaphone.fm/adchoices

The morning after the FDA's vaccine regulator Dr. Vinay Prasad announced his resignation, FDA Commissioner Dr. Marty Makary shares the agency's position on the measles vaccine, Sarepta's Duchenne therapy, and the addictive kratom-derived compound OH-7. Booking Holdings CEO Glenn Fogel discusses the impact of geopolitics on travel to the U.S. While Canadians and Europeans are still traveling, the operator of Agoda, Priceline, and Kayak says they're going to Mexico and Asia, instead of the U.S. Plus, Wall Street remembers the lives lost in Monday's shooting, the first tsunami waves from Russia's 8.8 earthquake have hit Hawaii's shores, and CNBC's Eamon Javers reports on his conversation with Treasury Secretary Scott Bessent about trade talks with China. Eamon Javers - 07:24Dr. Marty Makary - 18:51Glenn Fogel - 36:48 In this episode:Eamon Javers, @EamonJaversJoe Kernen, @JoeSquawk Becky Quick, @BeckyQuickAndrew Ross Sorkin, @andrewrsorkinKatie Kramer, @Kramer_Katie

The Sarepta saga continued into another week as the FDA recommended that the voluntary hold on the company's Duchenne muscular dystrophy gene therapy be lifted for ambulatory patients, after determining that the death of an 8-year-old Brazilian Duchenne patient who had received Elevidys' was not caused by the drug.  Sarepta's stock has swung wildly and its transparency questioned after it elected not to reveal the death of a third patient—a participant in a trial of a gene therapy for limb girdle muscular dystrophy—during a business update last week.    Speaking of entities—or individuals—who have trouble staying out of the news, Health Secretary Robert F. Kennedy Jr. plans to dissolve the U.S. Preventive Services Task Force because it is “too woke.” Also on Monday, Kennedy addressed what he called the “broken” vaccine injury compensation program. Without offering details, he vowed to “fix” the U.S.'s VICP and return it to its “original Congressional intent.”   On the business side of the biopharma house, Q2 earnings are in full swing, with AstraZeneca announcing estimate-beating numbers and CEO Pascal Soriot saying the world “needs to share” in global pharma R&D, while Merck cut $3 billion to support an aggressive launch schedule. Meanwhile, a week ahead of its own earnings report, Novo Nordisk named a new CEO and lowered its 2025 sales guidance for the second time this year.   In clinical development, the Alzheimer's Association Annual Conference is underway in Toronto, with Roche's trontinemab the standout so far. In a Phase Ib/IIa trial, the next-gen anti-amyloid antibody rapidly cleared amyloid from the brains of patients with Alzheimer's disease after just seven months—besting the 18-month timeframe for Biogen and Eisai's Leqembi and Eli Lilly's Kisunla. While Leqembi and Kisunla have shown some progress is slowing down the progression of Alzheimer's, their effect size is modest and they don't work for all patients—leaving plenty of room for symptomatic treatments, such as those being developed by Bristol Myers Squibb and Acadia Pharmaceuticals. The space is gearing up for several readouts, for both symptomatic and disease-modifying therapies alike.   And in BioPharm Executive this week, we dig into the top VC rounds so far this year and highlight a few scrappy biotechs walking the solo road.  

Sarepta Therapeutics (SRPT) jumped on Tuesday's session after the FDA lifted a voluntary pause on the company's gene therapy treatment for patients with Duchenne muscular dystrophy. The drug was pulled earlier this year to determine if it was tied to a patient's death. George Tsilis talks about the possibility of this being an "inflection bottom" for the stock, though analysts remain divided on whether that's actually the case.======== 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

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. The European Medicines Agency's CHMP did not recommend approving Elevidys for ambulatory patients with Duchenne muscular dystrophy, dealing a blow to Sarepta. FDA is rumored to request new data for Elevidys, leading to uncertainty as FDA considers a new study for the drug. In other news, Eli Lilly commits $856 million to Gate Bioscience for a new class of medicines, while Rocket trims headcount and pipeline focus. Roche also drops an early obesity asset as layoffs continue in the biopharma industry, with companies like Adicet optimizing their pipelines. Novartis makes a billion-dollar drug discovery deal with Matchpoint, and the FDA opens a pilot run of the commissioner voucher program.Stay tuned for more updates on the latest developments in the pharmaceutical and biotech world.

Il y a un an, Paris vibrait au rythme des Jeux olympiques et paralympiques. Depuis, la flamme s'est éteinte, mais que reste-t-il de cet été hors norme ? Avec une série de reportages, RFI revient pendant deux semaines sur les promesses, les transformations et les traces laissées par les Jeux dans la ville et dans les vies. Le département de la Seine-Saint-Denis commence à profiter des infrastructures construites durant cet événement. L'objectif en effet était de laisser un héritage aux habitants de ce département sous-doté en matière d'équipements sportifs. Des infrastructures emblématiques notamment avec le Centre aquatique olympique à Saint-Denis, qui a accueilli certaines épreuves, et le pôle de référence inclusif et sportif métropolitain (Prisme) à Bobigny, un lieu dédié principalement aux sportifs en situation de handicap. Après le dévoilement des anneaux, le Centre aquatique olympique-Métropole du Grand Paris, version héritage des Jeux, vient d'ouvrir ses portes au grand public. Viviane Gaessler, professeur de natation, encadre les élèves du collège Federico Garcia Lorca de Saint-Denis. « Ce sont des sixièmes, donc une tranche d'âge de 12 à 13 ans. On est dans l'apprentissage », décrit-elle. D'après la dernière enquête « Savoir nager », un enfant sur deux en Seine-Saint-Denis ne sait pas nager quand il arrive en sixième. « On s'était donné comme objectif d'apprendre à nager à la jeunesse de la Seine-Saint-Denis, un territoire dans lequel le taux d'apprentissage de la natation était nettement inférieur à la moyenne nationale. Avec ce centre aquatique et les autres bassins, on contribue à aller de l'avant pour apprendre à nager. On aura peut-être des petits champions dans les années à venir qui auront commencé ici, à la piscine, à Saint-Denis, dans le centre aquatique », se félicite Marie Barsacq, la ministre des Sports. « On a un dojo, une salle d'armes, une salle de danse, une salle de musculation et tout un espace de balnéothérapie. On a quand même 13 000 m2 de terrain de jeu à Bobigny. », énumère une jeune femme, qui visite le pôle de référence inclusif et sportif métropolitain, Prisme. « Aujourd'hui, on en récolte les fruits » À Bobigny, le Prisme, cet imposant bâtiment dans son enveloppe de dentelle blanche, ne passe pas inaperçu. Quatre mois après son ouverture, il accueille la Coupe du monde des clubs de foot fauteuil. « Ce lieu a été vraiment bien pensé pour les personnes en situation de handicap », se réjouit Erwan Conq, touché par la myopathie de Duchenne, capitaine de l'équipe de foot fauteuil de Châtenay-Malabry, qui découvre le lieu. « Ce site fait suite aux Jeux paralympiques, c'est quelque chose d'important. Je pense que cela va laisser une empreinte par rapport à ce qui a eu lieu il y a un an. Aujourd'hui, on en récolte les fruits. Nous qui sommes une discipline peu connue en tout cas, on se sert de ce lieu pour mettre en avant notre sport », estime-t-il. Luc, onze ans, fait ses débuts dans cette discipline. Il est heureux d'assister pour la toute première fois à cette compétition sportive avec sa mère. « On est au Prisme tous les samedis après-midi. On accueille déjà des jeunes en situation de handicap et on commence des initiations au foot fauteuil. J'ai les larmes qui me montent aux yeux de pouvoir assister à ce type de spectacle. Parce que pour moi, c'est vraiment un spectacle », confie-t-elle, émue. Le Prisme et le Centre aquatique olympique donnent une nouvelle image et redynamisent le département de la Seine-Saint-Denis, l'un des plus défavorisés de France métropolitaine. À lire aussiJO de Paris: un an après, le développement de la pratique du sport freiné par les coupes budgétaires

S&P Futures are displaying positive action this morning. Earnings report remain a key focus today as markets prepare for a heavy dose of tech earnings next week. President Trump visited the Fed yesterday in an effort to sway the Fed to lower interest rates. Today the President is headed to Scottland for personal business-related activities. Late yesterday afternoon, the Trump administration gave approval for CVX to resume oil production in Venezuela. The FCC approved the PARA merger with Skydance. SRPT is falling on concerns related to its Duchenne muscular dystrophy treatment. Next week is one of the busiest weeks of the year for markets. Highlights include a Fed meeting, US / China trade talks, key economic data points including GDP, PCE & Employment data, earnings from META, MSFT & AAPL, and expectations of trade deal announcements. This morning's key economic data point will be the report on Durable Goods Orders. On the earnings front, BAH, BYD, DECK, EW and MHK are higher after their releases. INTC, and LEA are moving lower.

Sarepta has temporarily paused shipments of Elevidys, its FDA-approved treatment for Duchenne muscular dystrophy. In this episode of GEN's Touching Base, we discuss the company's response to the tragedies associated with its DMD therapy as well as with a new therapy for limb-girdle muscular dystrophy. Also in this episode, big updates from AstraZeneca including a $50 billion investment in U.S. manufacturing and R&D, a heartwarming story about preventing mitochondrial disease involving eight babies from the U.K., and from the lab of Nobel Prize winner David Baker, PhD, AI that designs drugs for previously “undruggable” proteins.Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, PhD, and Uduak Thomas for a discussion of the latest biotech and biopharma news.Listed below are links to the GEN stories referenced in this episode of Touching Base:About Face: Sarepta to Pause Elevidys Shipments TemporarilyBy Alex Philippidis, GEN Edge, July 21, 2025StockWatch: Sarepta Shares Nosedive after LGMD Gene Therapy Patient DiesBy Alex Philippidis, GEN Edge, July 20, 2025Sarepta Axes 500, 36% of Workforce, in Restructuring after DMD Patient DeathsBy Alex Philippidis, GEN Edge, July 16, 2025AstraZeneca Commits $50B More to U.S. Manufacturing, R&D ProjectsBy Alex Philippidis, GEN Edge, July 22, 2025AstraZeneca's New $300M Plant Provides Control of Cell Therapy ProductionBy Gareth John Macdonald, GEN, May 14, 2025 Beyond Baby KJ: Next Steps in Manufacturing Genome Editing CuresGEN Live, July 30, 2025Mitochondrial Disease Milestone: Eight Babies Born Free of Disease via Pronuclear TransferBy Julianna LeMieux, PhD GEN, July 16, 2025Undruggable No More: AI Hits Disordered Proteins, Unlocks Therapy TargetsBy Fay Lin, PhD GEN, July 18, 2025 Hosted on Acast. See acast.com/privacy for more information.

Howie and Harlan are joined by public health communicator Katelyn Jetelina for updates on COVID-19 and other issues, and to discuss how her emails to students and colleagues in the early days of the pandemic turned into a platform with global reach. Harlan looks at how AI is being used on both sides of the battle between providers and insurers over claims; Howie reports on a setback with a promising gene therapy for Duchenne muscular dystrophy. Links: Insurance Claims, AI, and Wearables “Elevance Health Reports Second Quarter 2025 Results” “Elevance lowers profit outlook, warns things will get worse for health insurers ““Blue KC wrongfully denied medical diagnoses, hospital alleges in AI-driven claims lawsuit” “Oscar Health cuts full-year guidance, estimates 2025 loss as ACA marketplace stumbles” Whoop “WHOOP Delivers Innovative Blood Pressure Insights for a Deeper Look at Your Well-Being” “Introducing Heart Screener: A smarter way to stay connected to your heart health” “Whoop says FDA is ‘overstepping its authority' with warning about blood pressure feature” FDA: WARNING LETTER, WHOOP, Inc. “RFK Jr. wants everyone to use wearables. What are the benefits, risks?” “Apple to Sell Watches With Blood-Oxygen Feature Disabled After Legal Setback” Your Local Epidemiologist Your Local Epidemiologist “Poll: Trust in Public Health Agencies and Vaccines Falls Amid Republican Skepticism” “Popular epidemiologist lays out future path of public health communication” Yale School of Public Health: PopHIVE Katelyn Jetelina: “NIH: The quiet engine of science is being dismantled” Katelyn Jetelina: “Covid-19 in pockets, sugar cane isn't better, ticks march on, rescission cuts (vs. everything else), bright spots, and more” Mayo Clinic: Norovirus infection Katelyn Jetelina: “The show must go on...” Katelyn Jetelina: “Harassment against scientists is out of control” Katelyn Jetelina: “Medicaid cuts: The how and why” CDC: H5 Bird Flu: Current Situatio CDC: Current Epidemic Trends (Based on Rt) for States” CDC: Measles Cases and Outbreaks Treating Duchenne Muscular Dystrophy Cleveland Clinic: Duchenne Muscular Dystrophy (DMD) “FDA approves Sarepta's Duchenne gene therapy for nearly all patients” “AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial” “Patient dies in Sarepta gene therapy trial, adding to safety concerns” “FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths” “In surprise reversal, Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy” “Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks” Learn more about the MBA for Executives program at Yale SOM. Email Howie and Harlan comments or questions.

Howie and Harlan are joined by public health communicator Katelyn Jetelina for updates on COVID-19 and other issues, and to discuss how her emails to students and colleagues in the early days of the pandemic turned into a platform with global reach. Harlan looks at how AI is being used on both sides of the battle between providers and insurers over claims; Howie reports on a setback with a promising gene therapy for Duchenne muscular dystrophy. Links: Insurance Claims, AI, and Wearables “Elevance Health Reports Second Quarter 2025 Results” “Elevance lowers profit outlook, warns things will get worse for health insurers ““Blue KC wrongfully denied medical diagnoses, hospital alleges in AI-driven claims lawsuit” “Oscar Health cuts full-year guidance, estimates 2025 loss as ACA marketplace stumbles” Whoop “WHOOP Delivers Innovative Blood Pressure Insights for a Deeper Look at Your Well-Being” “Introducing Heart Screener: A smarter way to stay connected to your heart health” “Whoop says FDA is ‘overstepping its authority' with warning about blood pressure feature” FDA: WARNING LETTER, WHOOP, Inc. “RFK Jr. wants everyone to use wearables. What are the benefits, risks?” “Apple to Sell Watches With Blood-Oxygen Feature Disabled After Legal Setback” Your Local Epidemiologist Your Local Epidemiologist “Poll: Trust in Public Health Agencies and Vaccines Falls Amid Republican Skepticism” “Popular epidemiologist lays out future path of public health communication” Yale School of Public Health: PopHIVE Katelyn Jetelina: “NIH: The quiet engine of science is being dismantled” Katelyn Jetelina: “Covid-19 in pockets, sugar cane isn't better, ticks march on, rescission cuts (vs. everything else), bright spots, and more” Mayo Clinic: Norovirus infection Katelyn Jetelina: “The show must go on...” Katelyn Jetelina: “Harassment against scientists is out of control” Katelyn Jetelina: “Medicaid cuts: The how and why” CDC: H5 Bird Flu: Current Situatio CDC: Current Epidemic Trends (Based on Rt) for States” CDC: Measles Cases and Outbreaks Treating Duchenne Muscular Dystrophy Cleveland Clinic: Duchenne Muscular Dystrophy (DMD) “FDA approves Sarepta's Duchenne gene therapy for nearly all patients” “AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial” “Patient dies in Sarepta gene therapy trial, adding to safety concerns” “FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths” “In surprise reversal, Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy” “Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks” Learn more about the MBA for Executives program at Yale SOM. Email Howie and Harlan comments or questions.

Just a few weeks ago, it seemed like Sarepta had weathered a spate of bad news, after two patients died from liver injuries from its Duchenne muscular dystrophy gene therapy Elevidys. Then came news of a third patient death. Last Wednesday, the company announced a major restructuring and 500-person layoff.  Then, in just a few days time, Sarepta Therapeutics went from enjoying a notable stock bump in response to that corporate update to its lowest price in nearly 10 years as it halts shipments of Elevidys. In addition to requesting the shipment hold, the FDA revoked the company's technology platform designation and paused all clinical trials for Sarepta's limb-girdle muscular dystrophy (LGMD) gene therapy. The turmoil was set in motion by media reports that a patient who received the LGMD treatment had died—a fact the company chose not to disclose during an investor call. In other news, the FDA's Center for Drug Evaluation and Research gets a new director in biotech veteran George Tidmarsh, also an adjunct professor of pediatrics and neonatology at Stanford University's School of Medicine. Tidmarsh enters the agency at a time of mass layoffs as well as voluntary departures. Meanwhile, Replimmune and Roche suffer FDA rejections as therapies from Otsuka/Lundbeck and GSK fail to earn adcomm support, as the bar for acceptable controls and demonstrations of efficacy continue to change under FDA commissioner Marty Makary and CBER director Vinay Prasad.  Finally, Big Pharmas continue to pump billions into U.S. manufacturing, with Biogen and AstraZeneca joining the list of companies to have made such pledges, pledging $2 billion and $50 billion, respectively. These latest announcements come as President Donald Trump reiterates that pharma-specific tariffs of up to 200% could come as soon as Aug. 1. 

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Sarepta Therapeutics experienced a significant drop in shares as the FDA considers requesting a halt to shipments of the Duchenne muscular dystrophy therapy Elevidys. This decision comes after a third patient death linked to the underlying platform. The company's stock plummeted by 37% on Friday afternoon as reports of the potential shipment stop circulated in the media. This news adds to the already turbulent week for Sarepta, which has been facing challenges in its gene therapy and siRNA biotech developments. ## Novartis announced positive results from a phase 3 trial of its drug Cosentyx in treating axial spondyloarthritis. The study showed that patients treated with Cosentyx had significant improvements compared to those on a placebo. These results further solidify Novartis' position in the market for treatments of autoimmune diseases. The company plans to submit the data to regulatory authorities for potential approval of this indication.Novartis revealed encouraging outcomes from a phase 3 trial of its drug Cosentyx for treating axial spondyloarthritis. The study demonstrated that patients who received Cosentyx experienced notable enhancements compared to those who were given a placebo. These findings strengthen Novartis' standing in the autoimmune disease treatment market. The company intends to present the data to regulatory bodies for potential approval of this indication.## AstraZeneca faced setbacks as it announced delays in delivering its COVID-19 vaccine doses to the EU. The company cited production issues as the cause of the holdup, leading to frustration among European officials. This news comes at a time when vaccine distribution is crucial in combating the ongoing pandemic, highlighting the challenges faced by pharmaceutical companies in meeting global demand for vaccines.AstraZeneca encountered obstacles when it disclosed delays in distributing its COVID-19 vaccine doses to the EU. The company attributed production problems as the reason for the delay, causing frustration among European officials. This development occurs during a critical period in vaccine distribution to combat the current pandemic, underscoring the difficulties pharmaceutical companies encounter in meeting worldwide vaccine demands.## Pfizer and BioNTech announced plans to test a third dose of their COVID-19 vaccine to assess its effectiveness against new variants of the virus. The study will involve participants who have already received two doses of the vaccine and will evaluate the immune response generated by a booster shot. This initiative reflects ongoing efforts by pharmaceutical companies to adapt their vaccines to combat emerging strains of the virus.Pfizer and BioNTech unveiled intentions to examine a third dose of their COVID-19 vaccine to determine its efficacy against new virus variants. The research will include individuals who have already been administered two doses of the vaccine and will assess the immune response produced by an additional shot. This undertaking demonstrates continuous endeavors by pharmaceutical firms to modify their vaccines in response to evolving virus mutations.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in the Pharma and Biotech world. Sarepta Therapeutics has recently experienced an 18% increase in its stock value following a significant business overhaul, which included staff layoffs and pipeline shifts. Analysts are cautiously optimistic about the company's future. At the same time, patients are advocating for access to Brainstorm Cell Therapeutics' ALS drug, Nurown, after promising results from an expanded access program. In other news, Novartis is in the process of reshoring its drug manufacturing operations in the US, a move that may take several years to complete. Additionally, a notable number of employees have departed from the FDA's Center for Drug Evaluation and Research amidst an overhaul by the Department of Health and Human Services. These developments underscore the continuous changes and challenges within the pharmaceutical industry. Sarepta Therapeutics recently announced a strategic overhaul, which involved cutting 500 staff members and shifting focus to sirna platform assets. This decision came after two patients passed away following treatment with its Duchenne muscular dystrophy gene therapy, Elevidys. The company has also added a black box warning for acute liver injury and failure to Elevidys as it pivots away from gene therapy programs.

This episode is the third in a series hosted by Molecular Therapy Editor-in-Chief Joseph Glorioso, PhD, that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past 25 years. In this episode, Dr. Glorioso will discuss the reviews, “Current trends in gene therapy to treat inherited disorders of the brain,” with author Beverly Davidson, PhD, The Children’s Hospital of Philadelphia, and “The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy,” with author Jeff Chamberlain, PhD, University of Washington School of Medicine. If you enjoy this deep dive into research that is critical to the field, check out two more ASGCT events happening this fall: Advancing Cell and Gene Therapies for Cancer and Breakthroughs in Targeted In Vivo Gene Editing. During these events – in October and November, respectively – you’ll be able to hear directly from researchers about their own work related to these topics, or you can submit your own abstract for possible presentation. We hope to see you there! Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Up to 3,500 FDA staffers received their final walking papers Monday after the U.S. Supreme Court found last week that the government is “likely to succeed” in arguing that its overhaul of HHS is “lawful.” Meanwhile, FDA Commissioner Marty Makary floated policy changes for the agency, including a proposal to lower prescription drug user fees for the next iteration of the program, and one to offer speedier reviews to companies willing to lower the cost of their drugs.  Last week, the regulator opened its cache of complete response letters (CRLs), offering transparency into the rationale behind more than 200 recent rejections for ultimately approved therapies, including those for Eli Lilly's Alzheimer's drug Kisunla and Sarepta's Duchenne muscular dystrophy (DMD) treatment Vyondys 53. The FDA did not, however, release the CRLs for two new rejections: those of therapies from Ultragenyx and Capricor Therapeutics in Sanfilippo syndrome type A and cardiomyopathy associated with DMD, respectively. It was an especially rough week for Ultragenyx, which also, along with partner Mereo BioPharma, released seemingly negative Phase II/III data for their osteogenesis imperfecta therapy.  On a more positive note, two bustling therapeutic spaces continue to see positive data. In obesity, Hengrui Pharma's Kailera Therapeutics–partnered dual GLP-1/GIP receptor agonist elicited 17.7% average weight loss in a pivotal Chinese trial. And the psychedelic therapeutics space is again generating excitement with two recent positive readouts in treatment-resistant depression. BioSpace took a deep dive into the market reaction to these readouts for Compass Pathways and Beckley Psytech and atai Life Sciences, and what exactly investors are looking for in a successful psychedelic therapy.  Finally, we examine the progress of AI biotech unicorns and kick off our series on women in biopharma with profiles on Mayo Venture Partner Audrey Greenberg and the all-female CEO/R&D tandem at Acadia Pharmaceuticals.  

When Una Ennis' 7 year old son Archie was diagnosed with Duchenne muscular dystrophy, a condition that causes his muscles to weaken, her plea for help was shared all across social media. She told us how she first began to notice that Archie was struggling with basic things like jumping and climbing the stairs and how their family has coped since receiving the news of his diagnosis. Incredibly, Una and all of Archie's Army have now raised over 1 million euro for advanced treatments that can help to extend his life expectancy and keep him mobile - But they're still a long way from the €3.5 million they need.If you can, please consider helping Una by donating here: https://www.gofundme.com/f/4s5t9y-please-help-our-boy This episode was sponsored by Rightstyle Furniture - RIGHT PRICE, RIGHT STYLE, RIGHT NOW. Visit rightstyle.ie

Hoy preguntas y respuestas. Hola, hola, bienvenidos a El Café Positivo, tu espacio para empezar el día con neuroemoción y buena energía. Hoy vamos a hablar de una sonrisa… pero no cualquier sonrisa: la sonrisa Duchenne.Esa que aparece cuando algo nos emociona de verdad. Cuando ves a tu hijo reír dormido. Cuando alguien te dice ‘lo logré, gracias a ti'. Cuando no hay máscaras, solo verdad emocional.¿Sabías que no todas las sonrisas son iguales? La ciencia lo confirma: tu cara puede sonreír… sin que tú estés feliz.Pero hay una sonrisa que es inconfundible. No se puede fingir. Se llama sonrisa Duchenne, y ocurre cuando se activan dos músculos al mismo tiempo: 1. El cigomático mayor, que sube las comisuras de los labios. 2. Y el orbicular del ojo, que genera esas arruguitas en las esquinas de los ojos… las famosas ‘patas de gallo' que tanto queremos borrar con cremas, pero que en realidad son marcas de momentos reales de felicidad. Esta sonrisa es una metaemoción. Un indicador de que estamos haciendo bien las cosas. Que estamos conectando, que dejamos una huella. Cuando alguien te sonríe con una Duchenne, su cerebro ha liberado dopamina, oxitocina, endorfinas. Es química pura de bienestar. Y aquí va la pregunta poderosa del día:¿A quién le generaste una sonrisa Duchenne esta semana? ¿Y quién te la provocó a ti?La felicidad no siempre es una carcajada. A veces, es un microsegundo de verdad emocional que se nota en la mirada.Hoy, te invito a mirar con más atención… y a regalar sonrisas que nazcan desde adentro.

We catch up with Professor Thorsten Langer!Transition of youth with Duchenne muscular dystrophy: What are the key challenges in transitioning youth with Duchenne muscular dystrophy to adult care, and how can we address them?Join us for another brilliant conversation with a brilliant researcher - live from the EACD / IAACD Conference 2025, in Heidelberg Germany!

En este episodio de Más Que Raras, tenemos el privilegio de conversar con el Dr. Carlos Bacino, un referente internacional en genética molecular y humana. Con una sólida formación académica y décadas de experiencia, el Dr. Bacino ha dedicado su carrera al diagnóstico y tratamiento de enfermedades genéticas raras. Actualmente dirige la Clínica del Síndrome de Angelman y el laboratorio de citogenética en Baylor Genetics, desde donde lidera esfuerzos pioneros en investigación clínica y atención personalizada. En esta entrevista, nos habla de los avances científicos en su campo, su compromiso con las familias afectadas y la importancia de construir puentes entre la ciencia y la comunidad. Una conversación profunda y esperanzadora que no se pueden perder.   Más información:  El proyecto Texome: https://www.texome.org/ Centro de Enfermedades No Diagnosticadas en Baylor College of Medicine: https://www.bcm.edu/research/research-centers/undiagnosed-diseases-center   Fundación para la terapéutica del síndrome de Angelman (FAST LATAM) https://cureangelman.lat/   En Catalyst Pharmaceuticals, una empresa biofarmacéutica comprometida a mejorar las vidas de los pacientes con enfermedades poco frecuentes, entendemos los desafíos únicos de afecciones como la Distrofia Muscular de Duchenne y el Síndrome Miasténico de Lambert-Eaton (LEMS). Guiados por nuestro compromiso profundo hacia el cuidado del paciente, priorizamos la accesibilidad, al brindar un paquete integral de servicios de apoyo diseñados para acceso ininterrumpido y asistencia continua. Reconocemos que cada proceso es personal y puede ser difícil. Por eso es que nos dedicamos a brindar mucho más que tratamiento: estamos a su lado para apoyarlo a cada paso del camino.  Para obtener más información, visite catalystpharma.com  Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook BloodStream on Twitter    

Send us a textMeet Oakley Olson, a rising star in the trail running world who's breaking barriers between collegiate track and mountain racing. Fresh from her NCAA steeplechase season and now a Trail Team Elite selection for 2025, Oakley joins us just days before tackling both the Vertical Kilometer and 23K races at Broken Arrow Skyrace.Oakley's story is remarkable – from showing up at her first trail camp in worn-out road shoes to finishing top-10 at the US Mountain Running Championships within months. Speaking from the Trail Team camp in Grand Lake, Colorado, she shares how this supportive community transformed her understanding of the sport. "I had no idea what Broken Arrow was, what a running vest was, or what trail running shoes were," she laughs, describing her bloody-legged introduction to mountain terrain just a year ago.When the conversation turns to race strategy, Oakley reveals a mature approach beyond her experience level. Drawing from her NCAA racing background, she plans to start conservatively at Broken Arrow rather than getting caught in the frantic early pace. "I'd rather be the hunter than the hunted," she explains, a strategy that served her well at Snowbird last year against seasoned professionals.Most powerful is Oakley's motivation – she runs for her two younger brothers who have Duchenne muscular dystrophy. Wearing a lime green ribbon on her shoes, she pushes through difficult moments by remembering their daily struggles. "When it got so hard, the only thing that got me through was thinking about my two younger brothers and how that's how they feel every day walking up a staircase," she shares with heartfelt emotion. Her family will be waiting at the aid station atop the Vertical Kilometer, making a potential Team USA qualification even more meaningful.Listen now to this inspiring conversation with an athlete whose genuine passion, strategic intelligence, and heartfelt motivation are quickly making her one of trail running's most compelling figures. The future of American mountain running looks brighter with Oakley Olsen on the scene.Follow James on IG - @jameslaurielloFollow the Steep Stuff Podcast on IG - @steepstuff_podUse code steepstuffpod for 25% off your cart at UltimateDirection.com! 

This week, BioSpace is at 50% power as Heather McKenzie and Jef Akst are off attending this year's BIO Conference in Boston. The half-team discusses this week's biggest news: the death of another patient who took Sarepta's Duchenne muscular dystrophy gene therapy Elevidys. The patient was a non-ambulatory teenager who experienced acute liver failure after receiving the gene therapy, which is the same cause of death for an Elevidys patient reported in March. Sarepta announced that it was halting treatments to non-ambulatory patients and on a media call discussed new steps in its therapeutic protocol for preventing further liver injuries.  Elsewhere, mergers and acquisitions are surging across biopharma. Eli Lilly picked up the gene editing company Verve Therapeutics for $1.3 billion, which helped bolster the gene editing space —particularly after Sarepta's report of the death. Supernus bought Sage Therapeutics for $795 million, five months after Sage rejected a smaller offer from Biogen.  BioNTech also got in on the dealmaking, buying its German rival—scientifically and in the courtroom—CureVac for about $1.25 billion. The deal seemed focused mostly on CureVac's early-stage cancer immunotherapy pipeline, but analysts were otherwise left scratching their heads on what BioNTech was getting for its money.  Last week on The Weekly the team discussed the sudden dismissal of the CDC's entire ACIP committee, and this week we have a new slate of members. The eight people replacing the 17 members that were removed last week include allies of HHS Secretary Robert F. Kennedy Jr., many of whom are vaccine skeptics who seem to share his skeptical view of vaccination in general.  

A DYING MAN JUST WANTS TO TRY I realize that we are ALL dying but my guest today is on a fast track none of us want to be on. Elijah is 23 years old and is fighting for his life. He was only six years old when he was diagnosed with a fatal and incurable disease – Duchenne muscular dystrophy. The disease has robbed Elijah of the ability to walk. He's losing the use of his hands and arms. He likely only has a few years to live. There's a potential genetic treatment for his condition that is sitting on the shelf, all because of government bureaucracy. It could save his life, and others like him. That's why Elijah is advocating for the Right to Try for Individualized Treatments. Elijah he has become a strong advocate for Right to Try for Individualized Treatments (which we also call Right to Try 2.0). There is a treatment that could help him, but government red tape is preventing him from accessing it. So he has made it his mission to advocate for treatments like these/He also started a nonprofit, wrote a book, consults for biotech companies, and is working on a screenplay about his story. Read this story about his journey here. He joins me at 12:30 to talk about it. Find out more about Right to Try by clicking here.

In this powerful episode, we explore the emotional and often unseen journey of a mother's love. Sheryl Marazzo is a force of nature—fierce, steady, and filled with a bright hope. Living a life woven with heartbreak and tragedy, her resilience and sheer will drove her to find happiness and peace right alongside the unimaginable. As a mom to three children—one living with a rare disease, one a rare disease carrier, and one battling addiction—she brings honesty, humor, and deep heart to a conversation about survival, sacrifice, and the unexpected ways we figure out how to keep going. A long-time advocate and nonprofit founder, Sheryl's story is framed by Duchenne muscular dystrophy, but beyond labels and medical conditions, hers is simply a story about being fully human.

In this episode, recorded on April 4th in the stunning Seychelles, I sit down with Stéphane Duchenne, General Manager of Constance Ephelia Resort, and Markus Ultsch-Unrath, the resort's Sustainability, Health, and Safety Manager. In this chat, we explore their recent milestone achievement — becoming the first recipient of the Platinum Award at the Sustainable Seychelles Recognition & Certification Ceremony. Our wide-ranging conversation reveals how sustainability at Constance Ephelia goes far beyond carbon offsets and recycling bins. As Stéphane eloquently puts it, it's a philosophy rooted in the land, sung by the local community, and seen in the eyes of children playing among the mangroves. as Markus says: “We're not just here to protect nature — we're here to pass it on.”

CME credits: 1.00 Valid until: 09-05-2026 Claim your CME credit at https://reachmd.com/programs/cme/new-frontiers-in-the-treatment-of-dmd-across-the-age-spectrum/32740/ Explore the latest treatment options, age-specific management strategies, and clinical trial data for patients with Duchenne muscular dystrophy (DMD). Through expert-led discussions and real-world patient cases, you'll gain practical insights to help you confidently navigate today's ever-evolving treatment landscape and deliver personalized, informed care.=

About Andrea Andrea is a follower of Jesus, wife of Shawn, and mom of two boys; Sam (who has Duchenne Muscular Dystrophy) and Zach. As a younger adult, she obtained her Bachelor of Science in Civil Engineering, but left the workforce to homeschool her kids 15 years ago. Her family plays a small part in the special needs ministry 'Joni and Friends' via volunteering for their Wheels for the World program and enjoying their annual family camps. She's blessed to be mentor and friend to newer homeschool moms and to younger moms who are navigating the joys and the hard messiness of this special needs life. About the Episode In this conversation, Andrea Duerkop shares her journey as a special needs parent, discussing the challenges and triumphs of raising her son Sam, who has Duchenne muscular dystrophy. She reflects on the emotional journey of receiving the diagnosis, the importance of community support, and the lessons learned through faith and resilience. Andrea emphasizes the significance of sibling dynamics, the feeling of being loved and chosen, and the impact of giving back through service. The conversation highlights the importance of finding joy amidst challenges and the power of community in navigating the complexities of special needs parenting. Related Links By the Brook            

Após descobrir por acaso fotos de Josias na câmera da tia, Natalia se apaixonou por ele, mesmo sabendo que ele era noivo. Quando ele terminou o noivado, eles se aproximaram, casaram e tiveram duas filhas, apesar das dificuldades causadas pela trombofilia. Anos depois, Natalia engravidou naturalmente de Artur, um menino, o que a deixou aterrorizada por causa da distrofia muscular de Duchenne, doença genética que matou seu irmão. Mesmo com exames negativos, o diagnóstico de Artur se confirmou aos 5 anos. Existe um remédio que trava o avanço da doença, mas custa 17 milhões de reais e, apesar da liminar judicial que garantiu o direito ao tratamento, ele ainda não foi entregue. A família vive em isolamento rigoroso, separada das filhas, esperando que a Justiça seja cumprida para que Artur tenha a chance de viver.❤️ Se você quer contar sua história, é só entrar em contato através do nosso e-mail: quemamanaoesquece@band.com.br ou pelas nossas redes sociais. Você é fã da Band FM? Então, inscreva-se no canal e ative o sininho para não perder nenhum vídeo da rádio que é líder no Ibope em São Paulo há mais de 9 anos.

Drug development activities focused on the rare, neuromuscular condition Duchenne muscular dystrophy have translated into functional improvements and an extension of life expectancy. Regenxbio is among several companies pursuing a gene therapy to treat Duchenne. Regenxbio believes both its microdystrophin—a truncated form of the dystrophin gene­ small enough to fit in a vector­—and the vector it is using, give it a competitive advantage over other efforts. We spoke Curran Simpson, CEO of Regenxbio, about the company's platform technology, the advantages he sees with its experimental DMD gene therapy, and how a partnership announced at the start of 2025 focused on a pair of other gene therapies provided a welcome alternative to tapping the capital markets.

In this powerful and deeply personal episode of Making Our Way, we sit down with Vivian, a single mother caring for her son who is living with Duchenne muscular dystrophy. She opens up about the emotional aspect of caregiving—the quiet moments of exhaustion, the light moments of joy, and how she digs deep to draw on strength—even when she doesn't have much left to give. This conversation isn't just about Duchenne. It's about love and a tender connection between a mom and her son. It's about every parent who's ever felt overwhelmed. Every caregiver who's ever hidden their pain. And every person who's ever had to keep it all together, even when they're falling apart.

Hoy nos enfocaremos en experimentar una emoción de bienestar, más que en la simple observación física, como el roce del aire al respirar o la relajación de un músculo. Exploraremos la sensación de bienestar que surge con la sonrisa interior (sonrisa de Duchenne). Al cultivarla con regularidad, fortaleceremos esta experiencia y facilitaremos que el cerebro la integre de forma natural. Con la práctica constante, esta sensación de bienestar puede extenderse incluso después de la meditación. La intensidad y duración dependerán del tiempo y la constancia dedicados. Al principio, el proceso puede sentirse algo mecánico, pero con el tiempo se volverá más natural y fluido.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Pharma industry tensions rise as Trump tariffs miss sector for now. FDA's interim CBER head appointed after key resignation. Xaira Therapeutics hires top AI academic. Novavax's COVID-19 shot review deadline missed by FDA. Trilink offers guide RNA for CRISPR workflow. Biotech sector sees investment surge. Beigene scraps candidate, Sarepta's gene therapy on hold in Europe. Biopharma professionals work long hours.In other news, there are safety concerns in Duchenne treatment. Democrats challenge Trump health cuts. And there are job opportunities in the biopharma industry.

Dr. Scott Younger is the Director of Disease Gene Engineering within the Genomic Medicine Center at Children's Mercy Hospital. His research focuses on producing patient-derived cellular models to develop functional precision medicine. He talks about using personalized antisense oligonucleotides to reverse disease phenotypes in organoid models of Duchenne muscular dystrophy. He also discusses his lab's personal connections to the rare disease community and the opportunities for collaborations with clinicians at Children's Mercy.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Sanofi and Alnylam have received FDA approval for the first RNAi treatment for hemophilia, with the drug, Qfitlia, indicated for both hemophilia A and B. This approval is significant as it can be given regardless of the presence of neutralizing antibodies against clotting factors VIII or IX. However, the sudden departure of FDA director Peter Marks has caused uncertainty in the biopharma industry. In other news, Vertex has cut a diabetes asset but analysts remain optimistic about their phase III option. Lilly's RNA silencer has shown promising results in lowering a key cardiovascular biomarker. Trilink is offering custom guide RNAs for CRISPR workflow to accelerate therapy discoveries. Despite market challenges, the cell and gene therapy sector has seen a 30% investment surge. Companies like Amgen, Aldeyra, and Argenx are among those with upcoming FDA actions. Arbutus has announced layoffs, while big pharmas are pushing boundaries in radiopharmaceuticals. Michelle Werner of AltoRNA is focused on making better drugs. Safety questions are looming in Duchenne as Dyne and Wave plan FDA filings. There are job opportunities available in data management and program leadership within the biopharma industry.Moving on to other news, several big pharmaceutical companies such as Novartis, Bayer, AstraZeneca, Bristol Myers Squibb, and Eli Lilly are competing in the radiopharmaceuticals market, which is projected to be worth over $13 billion by 2033. The FDA is expected to announce decisions on therapies for dry eye disease soon. Michelle Werner, CEO of AllTrna, is focused on developing trna-based treatments for various diseases.Safety concerns are emerging in the Duchenne muscular dystrophy space as companies like Dyne and Wave plan FDA filings. The EU rejected Lilly's Alzheimer's drug Kisunla, Biontech's bispecific showed promise in treating SCLC patients, and Wave's duchenne exon-skipper reversed muscle damage in a mid-stage trial. Job opportunities within the biopharma industry were also highlighted for those interested.Thank you for tuning in to Pharma and Biotech daily - keeping you updated on all the latest news in the world of pharmaceuticals and biotechnology.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Sanofi has committed up to $1.9 billion to acquire Dren Bio's bispecific antibody for autoimmune disease, adding to its investments in the immunology portfolio. The deal comes after the tragic death of a patient who had taken the gene therapy Elevydys, prompting the Duchenne patient community to vow to push on. Paratek Pharmaceuticals has acquired Optinose for up to $330 million, while Senate Democrats demand the return of fired CDC staff. Sino Biological has developed recombinant antigens for the 2025-2026 influenza vaccine strains, and Purdue has filed for bankruptcy to support a $7.4 billion opioid settlement. Doctors continue to rally behind vaccines amidst doubts and misinformation, and Novartis' intrathecal Zolgensma has shown effectiveness in older children. TC Biopharm and Cargo have enacted steep workforce reductions. Pharmaceutical companies are also preparing for upcoming events, including webinars on AI regulation and drug development. Job opportunities in the pharmaceutical industry are available at companies like Takeda, Eli Lilly and Company, and Novo Nordisk.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.## Sarepta's DMD gene therapy patient dies, causing a 22% drop in shares.Sarepta experienced a significant setback as a patient participating in their DMD gene therapy trial tragically passed away, leading to a 22% decrease in their shares.## Arbor raises $73.5 million for gene editing research.Arbor successfully secured $73.5 million in funding to further their research in gene editing, showcasing the growing interest and investment in this innovative field.## Opko and Entera collaborate on oral GLP-1/glucagon drug development.Opko and Entera have joined forces to collaborate on the development of an oral GLP-1/glucagon drug, combining their expertise to potentially revolutionize treatment options for patients.## Mirador aims to be a leading I&I breakthrough by 2030.Mirador has set ambitious goals to become a leading breakthrough in the field of inflammation and immunology by the year 2030, highlighting their commitment to advancing healthcare solutions.## Sino Biological offers reagents for 2025-2026 influenza vaccine strains.Sino Biological is providing reagents for the upcoming 2025-2026 influenza vaccine strains, contributing to the global efforts to combat infectious diseases and protect public health.## AstraZeneca invests $1.35 billion in subcutaneous cancer drugs with Alteogen alliance.AstraZeneca has made a substantial investment of $1.35 billion in subcutaneous cancer drugs through a strategic alliance with Alteogen, signaling their dedication to advancing oncology treatments.## DYNE's Duchenne exon skipping oligomer shows promising clinical effect.DYNE's Duchenne exon skipping oligomer has demonstrated promising clinical effects, offering hope for patients with this debilitating genetic disorder and potentially paving the way for new treatment options.Thank you for tuning in to Pharma and Biotech daily, where we bring you the latest updates and developments shaping the pharmaceutical and biotechnology industries. Stay informed and stay ahead in the world of healthcare innovation.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.AstraZeneca has recently made a significant investment of up to $1 billion in cell therapy through the acquisition of esobiotec. This move is part of AstraZeneca's larger strategy to expand in the cell therapy space, positioning itself to be a major player in the market. Dyne is also looking to file for accelerated approval for its Duchenne exon skipping oligomer, while Taiho Pharmaceutical has acquired ADC partner Araris for up to $1.1 billion. Alnylam is expected to enter the transthyretin amyloid cardiomyopathy market, which is projected to reach $11.2 billion by 2030.Sino Biological has developed reagents for the 2025-2026 influenza vaccine strains, offering a range of recombinant proteins for vaccine development. The pharmaceutical industry is seeing significant activity in various therapy areas, with companies making strategic investments and advancements in research and development.Alnylam is awaiting approval for their drug Amvuttra in the transthyretin amyloid cardiomyopathy market, which is currently dominated by Pfizer and BridgeBio. The obesity drug market is becoming increasingly competitive, with companies focusing on overall health benefits rather than just weight loss. Biopharma companies are now exploring the use of CAR T cell therapies for autoimmune disorders, with several readouts expected this year. Ionis and Ultragenyx are competing to develop treatments for Angelman syndrome, while Neuren is trying to catch up.Overall health outcomes are becoming more important in the obesity drug market, with successful studies in therapeutic areas like cardiovascular and sleep apnea providing a market advantage. Other news includes flu vaccine recommendations from the FDA and updates on drugs for alcohol use disorder, plaque psoriasis, breast cancer, and weight loss. The biopharma industry continues to evolve, with readers encouraged to provide suggestions for future coverage topics.

Llegan al IECM listados de  candidatos a elección judicial INR revela placa como el primer Centro Duchenne certificado en México Zelensky es incapaz de llegar a acuerdos: Rusia Más información en nuestro podcast

Organoids, three-dimensional cell models that can replicate an individual's organs, are valuable tools for testing medicines that might treat their illness. It can, however, take up to $10,000 and a year to grow organoids using conventional methods from patient-derived induced pluripotent stem cells. Researchers at Children's Mercy Kansas City's Genomic Medicine Center developed a way to do this from about $200 and in two to three weeks. We spoke to Scott Younger, director of disease gene engineering at Children Mercy Kansas City's Genomic Medicine Center, about the process, the test it ran to match three children with Duchenne muscular dystrophy to an antisense oligonucleotide therapy, and the potential impact this may have on developing customized therapies for people with rare genetic diseases.

Clinical-stage drug development offers big rewards—and big risks. To that end, Fierce Biotech recently published its annual roundup of several of the most eye-catching trial failures of the preceding year. The 2024 list includes trial flops from the likes of AbbVie, Novo Nordisk, Pfizer and more, with reports of disappointing results in many tough-to-treat indications, including schizophrenia and Alzheimer's disease. In this week’s episode of The Top Line, we dive into the report. Fierce Biotech’s James Waldron and Gabrielle Masson discuss the entries that stood out for them and ask what lessons the biopharma industry can learn from these setbacks going forward. To learn more about the topics in this episode: 2024's top 10 clinical trial flops AbbVie's $9B schizophrenia prospect flunks phase 2 trials, handing advantage to BMS GSK surrenders HSV vaccine hopes after phase 2 fail, ceding race to Moderna, BioNTech Merck halts phase 3 TIGIT trial after immune-mediated adverse events prompt discontinuations Pfizer's phase 3 gene therapy trial fails to improve function for boys with Duchenne muscular dystrophy See omnystudio.com/listener for privacy information.

What if the treatment they said was impossible… actually worked? In this inspiring episode of The Real Health Podcast, Dr. Ron Hunninghake, MD sits down with Blake Benton to discuss his family's incredible journey with stem cell therapy for Duchenne muscular dystrophy. Learn how Coming Together for a Cure, a nonprofit founded by the Benton family, is now helping hundreds of families access life-changing treatments!Learn more about our guest, Blake Benton:•Coming Together for a Cancer Cure: https://www.ctfac.org/•Watch The Sunshine Dreamer Documentary: https://www.ctfac.org/the-sunshine-dreamer-documentaryLearn more about the hosts:Dr. Ron Hunninghake, MD: https://riordanclinic.org/staff/ron-hunninghake-md/Interested in becoming a Patient:https://riordanclinic.org/request-an-appointment/Read the transcript:https://realhealthpodcast.orgLearn more about Riordan Clinic:https://riordanclinic.org/Thanks to This Episode's SponsorRiordan Clinic Nutrient Store: https://store.riordanclinic.org/Disclaimer: The information contained on the Real Health Podcast and the resources mentioned are for educational purposes only. They're not intended as and shall not be understood or construed as medical or health advice. The information contained on this podcast is not a substitute for medical or health advice from a professional who is aware of the facts and circumstances of your individual situation. Information provided by hosts and guests on the Real Health Podcast or the use of any products or services mentioned does not create a practitioner-patient relationship between you and any persons affiliated with this podcast.

Discover the transformative power of the pivot with Andrea Miller. After her son was diagnosed with Duchenne muscular dystrophy, Andrea faced a life-altering challenge that reshaped her mindset and approach to change. Guided by her faith in God and her inspiring mantra, “Today, Not Tomorrow” (TNT), Andrea found the strength to navigate life's uncertainties with purpose and courage. In this episode, Andrea shares her remarkable journey, revealing the strategies, lessons, and moments of trial and error that helped her thrive. From asking the right questions to embracing imperfection, her insights are a masterclass in resilience and growth.Whether you're navigating a career shift, starting a new venture, or seeking the confidence to take bold steps forward, Andrea's practical advice and motivational stories will inspire you to move from feeling stuck to truly thriving.Hit play and let Andrea's story empower you to embrace change fearlessly and make the most of today.CONNECT WITH ANDREA MILLERSend us a textFor more info on IMAGINE YOURSELF, visit imagineyourselfpodcast.com. You'll find blogs, inspirational quotes and of course our podcasts!Join the conversation on our FACEBOOK, or INSTAGRAM pages. Email at imagineyourselfpodcast@gmail.com Thanks for being part of the Imagine Yourself Family! Follow or subscribe so you don't miss an episode!Imagine Yourself is hosted by Lanée Blaise and Sandy Kovach. Lanée is a TV writer and producer, motivational speaker and podcaster. Sandy is a radio personality, voiceover artist and podcaster. They come to you from the Detroit Metro area and invite guests from all over the world to help encourage you in your health, career, faith journey and more!

  It's hard to fathom why certain children receive life altering diagnoses. Pediatricians simply want to help, and that help usually comes in the form of early diagnosis. Duchenne muscular dystrophy, otherwise known as Duchenne or DMD, is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births. Early screening and identification can mean quicker access to care, early intervention programs and special education resources. Thanks to early diagnosis and advances in care, life expectancy is increasing.  Our guest joining us to discuss diagnosing and treating DMD is Michele Yang, MD, who specializes in pediatric neurology and neuromuscular medicine at Children's Hospital Colorado. She is also an associate professor of pediatric neurology at the University of Colorado School of Medicine.  Some highlights from this episode include:  How diagnosis of DMD has changed over the years.  Understanding the new, advanced treatments that exist.  Tips and tricks for primary care providers to utilize.  Realistic outcomes for these kids with DMD.  For more information on Children's Colorado, visit: childrenscolorado.org.