Podcasts about Duchenne

Author and screenwriter Sai Marie Johnson flatlined during a high-risk C-section complicated by placenta percreta. While doctors fought to stop catastrophic hemorrhaging, Sai experienced a profound “void, tunnel and light." During her NDE (Near Death Experience) she met and saw her deceased grandmother and great-grandmother. They told her that she had to go back. Above all else, she felt "Every good emotion we can feel on earth, times 5,000!" Goodness and Pure Love. Following her NDE, she woke to a premature son in the NICU, a partial hysterectomy, and 25 transfusions—but also a new clarity that reshaped her confidence, career, and view of death. In this conversation, she unpacks the medical rollercoaster, the otherworldly peace she felt, how she parents two sons with Duchenne muscular dystrophy, and the hard-won faith that guides her now. VIDEO VERSION: https://youtu.be/nGlyLx3LDVwChapters (Timestamps)00:00 — Cold Open: “They pronounced me dead…”00:40 — Welcome & Guest Intro: Meet author/screenwriter Cy Marie Johnson01:19 — About Cy: Mother of four; two sons with Duchenne03:23 — High-Risk Pregnancy: Early warnings and specialist care04:40 — From Previa to Percreta: What went medically wrong06:08 — Hospital Prep: Steroids, IVs, arterial balloons, central line10:41 — Countdown to Two: Going under anesthesia11:01 — The Experience: Darkness, distant ember, irresistible light12:04 — The Guardians: Grandmother & great-grandmother say “not yet”13:52 — Void or Tunnel? How it felt, and what drew her forward15:11 — Who They Were: Portraits of Elizabeth and Bernice19:31 — Back in the Body: “Where's my baby?” — NICU update22:15 — Recovery Begins: Hysterectomy, pain, and walking laps24:51 — What Happened on the Table: Hemorrhage and 25 transfusions26:13 — Life After NDE: Confidence, creativity, a second chance28:49 — Writing Career: From romance to nonfiction craft29:57 — Duchenne Awareness: Strength for her sons30:44 — On Death: Why she isn't afraid32:14 — Closing & Call to ActionRoundTripDeath.comDonate to this podcast: https://www.roundtripdeath.com/support/

Send us a textCheck our the full viva in the Final Exam Coursehttps://anaesthesia.thinkific.com/courses/FinalExam---------Find us atInstagram: https://www.instagram.com/abcsofanaesthesia/Twitter: https://twitter.com/abcsofaWebsite: http://www.anaesthesiacollective.comPodcast: ABCs of AnaesthesiaPrimary Exam Podcast: Anaesthesia Coffee BreakFacebook Page: https://www.facebook.com/ABCsofAnaesthesiaFacebook Private Group: https://www.facebook.com/groups/2082807131964430---------Check out all of our online courses and zoom teaching sessions here!https://anaesthesia.thinkific.com/collectionshttps://www.anaesthesiacollective.com/courses/---------#Anesthesiology #Anesthesia #Anaesthetics #Anaesthetists #Residency #MedicalSchool #FOAMed #Nurse #Medical #Meded ---------Please support me at my patreonhttps://www.patreon.com/ABCsofA---------Any questions please email abcsofanaesthesia@gmail.com---------Disclaimer: The information contained in this video/audio/graphic is for medical practitioner education only. It is not and will not be relevant for the general public.Where applicable patients have given written informed consent to the use of their images in video/photography and aware that it will be published online and visible by medical practitioners and the general public.This contains general information about medical conditions and treatments. The information is not advice and should not be treated as such. The medical information is provided “as is” without any representations or warranties, express or implied. The presenter makes no representations or warranties in relation to the medical information on this video. You must not rely on the information as an alternative to assessing and managing your patient with your treating team and consultant. You should seek your own advice from your medical practitioner in relation to any of the topics discussed in this episode' Medical information can change rapidly, and the author/s make all reasonable attempts to provide accurate information at the time of filming. There is no guarantee that the information will be accurate at the time of viewingThe information provided is within the scope of a specialist anaesthetist (FANZCA) working in Australia.The information presented here does not represent the views of any hospital or ANZCA.These videos are solely for training and education of medical practitioners, and are not an advertisement. They were not sponsored and offer no discounts, gifts or other inducements. This disclaimer was created based on a Contractology template available at http://www.contractology.com.

El C.D. Leganés lanzaba un llamamiento esta semana para ayudar a un joven aficionado pepinero, Uriel, que padece distrofia muscular de Duchenne, y que, tras sufrir una caída y romperse la cadera, necesita de una silla de ruedas especial para poder recuperarse.

Which combination of impairments is MOST likely to be present in a 7-year-old patient with Duchenne muscular dystrophy? Find it all out in the podcast! Be prepared for the NPTE so that you can pass with flying colors! Check out www.ptfinalexam.com/podcast for more information and to stay up-to-date with our latest courses and projects. #Npte #PT #ptboards #crushtheNPTE #study #studygram #spt #ptstudent #ptlife #sptprobs #physicaltherapystudent #physicaltherapy #physio #physiotherapist #ptlife #ptstudentstudy

Limb-girdle muscular dystrophies (LGMDs) encompass a group of genetically heterogeneous skeletal muscle disorders. There has been an explosion of newly identified LGMD subtypes in the past decade, and results from preclinical studies and early-stage clinical trials of genetic therapies are promising for future disease-specific treatments. In this episode, Gordon Smith, MD, FAAN, speaks with Teerin Liewluck, MD, FAAN, FANA, author of the article “Limb-Girdle Muscular Dystrophies” in the Continuum® October 2025 Muscle and Neuromuscular Junction Disorders issue. Dr. Smith is a Continuum® Audio interviewer and a professor and chair of neurology at Kenneth and Dianne Wright Distinguished Chair in Clinical and Translational Research at Virginia Commonwealth University in Richmond, Virginia. Dr. Liewluck is a professor of neurology at the Division of Neuromuscular Medicine and Muscle Pathology Laboratory at Mayo Clinic College of Medicine in Rochester, Minnesota. Additional Resources Read the article: Limb-Girdle Muscular Dystrophies Subscribe to Continuum®: shop.lww.com/Continuum Earn CME (available only to AAN members): continpub.com/AudioCME Continuum® Aloud (verbatim audio-book style recordings of articles available only to Continuum® subscribers): continpub.com/Aloud More about the American Academy of Neurology: aan.com Social Media facebook.com/continuumcme @ContinuumAAN Host: @gordonsmithMD Guest: @TLiewluck Full episode transcript available here Dr Jones: This is Dr Lyell Jones, Editor-in-Chief of Continuum. Thank you for listening to Continuum Audio. Be sure to visit the links in the episode notes for information about earning CME, subscribing to the journal, and exclusive access to interviews not featured on the podcast. Dr Smith: This is Dr Gordon Smith with Continuum Audio. Today I'm interviewing Dr Teerin Liewluck, a good friend of mine at the Mayo Clinic, about his article on the limb girdle muscular dystrophies. This article appears in the October 2025 Continuum issue on muscle and neuromuscular junction disorders, a topic that is near and dear to my heart. Teerin, welcome to the podcast, and maybe you can introduce yourself to our listeners. Dr Liewluck: Thank you very much, Gordon, and I want to say hi to all the Continuum fans. So, I'm Dr Teerin Liewluck, I'm the professor of neurology at Mayo Clinic in Rochester, Minnesota. So, my practice focus on all aspects of muscle diseases, both acquired and genetic myopathies. Glad to be here. Dr Smith: I just had the great pleasure of seeing you at a seminar in Houston where you talked about this topic. And so, I'm really primed for this conversation, which I'm very excited about. I find this topic a little hard, and I'm hoping I can learn more from you. And I wonder if, as we get started, recognizing many of our listeners are not in practices focused purely on muscle disease, maybe you can provide some context about why this is important for folks doing general neurology or even general neuromuscular medicine? Why do they need to know about this? Dr Liewluck: Yes, certainly. So, I would say limb girdle muscular dystrophy probably the most complex category of subgroup of muscle diseases because, by itself, it includes thirty-four different subtypes, and the number's still expanding. So, each subtype is very rare. But if you group together, it really have significant number of patients, and these patients present with proximal weakness, very high CK, and these are common patients that can show up in the neurology clinic. So, I think it's very important even for general neurologists to pick up what subtle clues that may lead to the diagnosis because if we are able to provide correct diagnosis for the patients, that's very important for patient management. Dr Smith: So, I wonder if maybe we can talk a little bit about the phenotype, Terran. I mean, your article does a great job of going over the great diversity. And you know, I think many of us here, you know, limb girdle muscular dystrophy and we think of limb girdle weakness, but the phenotypic spectrum is bananas, right? Rhabdomyolysis, limb girdle distal myopathy. I mean, when should our listeners suspect LGMD? Dr Liewluck: Yes, I think by the definition to all the LGMD patients will have limb girdle of proximal weakness and very high CK. So, these are common phenotypes among thirty-four different subtypes. But if it did take into details, they have some subtle differences. In the article, what I try to simplify all these different subtypes that we can categorize at least half of them into three main group that each group the underlying defect sharing among those subtypes and also translate into similar muscles and extra muscular manifestations. You will learn that some of the limb girdle muscular dystrophy may present with rhabdomyolysis. And we typically think of this as metabolic myopathies. But if you have a rhabdomyolysis patient, the CK remain elevated even after the acute episode, that's the key that we need to think this could be LGMD. That's for an example. Dr Smith: So, I wonder if maybe we can start there. I was going to go in a different direction, but this is a good transition. It's easy to see the opportunity to get confused between LGMD or, in that case, a metabolic myopathy or other acquired myopathies. And I think particularly adult neurologists are more accustomed to seeing acquired muscle disease. Are there particular clues that, or pearls that adult neurologists seeing patients with muscle disease can use to recognize when they should be thinking about LGMD given the diverse phenotype? Dr Liewluck: Yes. What I always tell the patient is that there are more than a hundred different types of muscle diseases, but we can easily divide into groups: acquired and genetic or hereditary. So, the acquired disease is when you encounter the patients who present with acute or subacute cause of the weakness, relatively rapidly progressive. But on the opposite, if you encounter the patient who present with a much more slowly progressive cause of weakness over several months or years, you may need to think about genetic disease of the muscle with also including limb-girdle muscular dystrophy. The detailed exam to be able to distinguish between each type of muscular dystrophy. For example, if proximal weakness, certainly limb girdle muscular dystrophy. If a patient has facial weakness, scapular winking, so you would think about facial scapular hematoma dystrophy. So, the slowly progressive cause of weakness, proximal pattern of weakness, CK elevation, should be the point when you think about LGMD. Dr Smith: So, I have a question about diagnostic evaluation. I had a meeting with one of my colleagues, Qihua Fan, who's a great peripheral nerve expert, who also does neuromuscular pathology. And we were talking about how the pathology field has changed so much over the last ten years, and we're doing obviously fewer muscle biopsies. Our way of diagnosing them has changed a lot with the evolution of genetic testing. What's your diagnostic approach? Do you go right to genetic testing? Do you do targeted testing based on phenotype? What words of wisdom do you have there? Dr Liewluck: Yes, so, I mean, being a muscle pathologist myself, it is fair to say that the utility of muscle biopsies when you encounter a patient with suspects that limb girdle muscular dystrophy have reduced over the year. For example, we used to have like fifteen, seventeen hundred muscle biopsies a year; now we do only thirteen hundred biopsies a year. Yes, as you pointed out, the first step in my practice if I suspect LGMD is to go with genetic testing. And I would prefer the last gene panel that not only include the LGMD, but also include all other genetic muscle disease as well as the conjunctive myopic syndrome, because the phenotype can be somehow difficult to distinguish in certain patients. Dr Smith: So, do you ever get a muscle biopsy, Teerin? I mean you obviously do; only thirteen hundred. Holy cow, that's a lot. So, let me reframe my question. When do you get a muscle biopsy in these patients? Dr Liewluck: Muscle biopsy still is present in LGMD patients, it's just we don't use it at the first-tier diagnostic test anymore. So, we typically do it in selected cases after the genetic testing in those that came back inconclusive. As you know, you may run into the variant of unknown significance. You may use the muscle biopsy to see, is there any histopathology or abnormal protein Western blot that may further support the heterogenicity of the VUS. So, we still do it, but it typically comes after genetic testing and only in the selected cases that have inconclusive results or negative genetic testing. Dr Smith: I'd like to ask a question regarding serologic testing for autoantibodies. I refer to a really great case in your article. There are several of them, but this is a patient, a FKRP patient, who was originally thought to have dermatomyositis based on a low-titer ME2 antibody. You guys figured out the correct diagnosis. We send a lot of antibody panels out. Wonder if you have any wisdom, pearls, pitfalls, for how to interpret antibody tests in patients with chronic myopathies? We send a lot of them. And that's the sort of population where we need to be thinking about limb-girdle muscular dystrophies. It's a great case for those, which I hope is everyone who read your article in detail. What do you have to say about that? Dr Liewluck: Yes, so myositis antibodies, we already revolutionized a few of muscle diseases. I recall when I finished my fellowship thirteen years ago, so we don't really have much muscle myositis antibodies to check. But now the panel is expanded. But again, the antibodies alone cannot lead to diagnosis. You need to go back to your clinical. You need to make sure the clinical antibodies findings are matched. For example, if the key that- if the myocytes specific antibodies present only at the low positive title, it's more often to be false positive. So, you need to look carefully back in the patient, the group of phenotypes, and when in doubt we need to do muscle biopsies. Now on the opposite end, the other group of the antibody is the one for necrotizing autoimmune myopathy; or, the other name, immune-mediated necrotizing myopathy. This is the new group that we have learned only just recently that some patients may present as a typical presentation. I mean, when even thinking about the whole testing autoimmune myopathy, we think about those that present with some acute rapidly progressive weakness, maybe has history of sudden exposures. But we have some patients that present with very slowly progressive weakness like muscular dystrophies. So now in my practice, if I encounter a patient I suspect LGMD, in addition to doing genetic testing for LGMD, I also test for necrotizing doing with myopathy antibodies at the same time. And we typically get antibody back within what, a week or two, but projected testing would take a few months. Dr Smith: Yeah. And I guess maybe you could talk a little bit about pitfalls and interpretation of genetic tests, right? I think you have another case in your article, and I've certainly seen this, where a patient is misdiagnosed as having a genetic myopathy, LGMD, based on, let's say, just a misinterpretation of the genetic testing, right? So, I think we need to think of it on both sides. And I like the fact that the clinical aspects of diagnosis really are first and foremost most important. But maybe you can talk about wisdom in terms of interpretation of the genetic panel?  Dr Liewluck:Yes. So genetic testing, I think, is a complex issue, particularly for interpretation. And if you're not familiar with this, it's probably best to have your colleagues in genetics that help looking at this together. So, I think the common scenario we encounter is that in those dystrophies that are autosomal recessive, so we expect that the patient needs to have two abnormal copies of the genes to cause the disease. And if patients have only one abnormal copy, they are just a carrier. And commonly we see patients refer to us as much as dystrophy is by having only one abnormal copy. If they are a carrier, they should not have the weakness from that gene abnormality. So, this would be the principle that we really need to adhere. And if you run into those cases, then maybe you need to broaden your differential diagnosis. Dr Smith: I want to go back to the clinical phenomenology, and I've got a admission to make to you, Teerin. And I find it really hard to keep track of these disorders at, you know, thirty-four and climbing a lot of overlap, and it's hard to remember them. And I'm glad that I'm now going to have a Continuum article I can go to and look at the really great tables to sort things out. I'm curious whether you have all these top of mind? Do you have to look at the table too? And how should people who are seeing these patients organize their thoughts about it? I mean, is it important that you memorize all thirty-four plus disorders? How can you group them? What's your overall approach to that? Dr Liewluck: I need to admit that I've not memorize all twenty-four different subtypes, but I think what I triy to do even in my real-life practice is group it all together if you can. For example, I think that the biggest group of these LGMD is what we call alpha-dystroglycanopathies. So, this include already ten different subtypes of recessive LGMD. So alpha-dystroglycan is the core of the dystrophin-associated glycoprotein complex. And it's heavy glycosylated protein. So, the effect in ten different genes can affect the glycosylation or the process of adding sugar chain to this alpha-dystroglycan. And they have similar features in terms of the phenotype. They present with proximal weakness, calf pseudohypertrophy, very high CK, some may have recurrent rhabdomyolysis, and cardiac and rhythmic involvement are very common. This is one major group. Now the second group is the limb-girdle muscular dystrophy due to defective membrane repair, which includes two subtypes is the different and on dopamine five. The common feature in this group is that the weakness can be asymmetric and despite proximal weakness, they can have calf atrophy. On muscle biopsy sometimes you can see a myeloid on the muscle tissues. And the third group is the sarcoglycanopathy, which includes four different subtypes, and the presentation can look like we share. For the rest, sometimes go back to the table. Dr Smith: Thank you for that. And it prompts another question that I always wonder about. Do you have any theories about why such variability in the muscle groups that are involved? I mean, you just brought up dystroglycanopathy, for instance, as something that can cause a very distal predominant myopathy; others do not. Do we at this point now have an understanding given the better genetics that we have on this and work going on in therapeutic development, which I want to get to in a minute, that provides any insight why certain muscle groups are more affected? Dr Liewluck: Very good question, Gordon. And I would say the first question that led me interested in muscle disease---and this happened probably back in 2000 when I just finished medical school---is why, why, why? Why does muscle disease tend to affect proximal muscles? I thought by now, twenty-five years later, we'd have the answer. I don't. I think this, you don't know clearly why muscle diseases, some affect proximal, some affect distal. But the hypothesis is, and probably my personal hypothesis is, that maybe certain proteins may express more in certain muscles and that may affect different phenotypes. But, I mean, dysferlin has very good examples that can confuse us because some patients present with distal weakness, some patients present with proximal weakness, that's by the same gene defect. And in this patient, when we look at the MRI in detail, actually the patterns of fatty replacements in muscle are the same. Even patient who present clinically as a proximal or distal weakness, the imaging studies show the same finding. Bottom line, we don't know. Dr Smith: Yeah, who knew it could be so complex? Teerin, you brought up a really great point that I wanted to ask about, which is muscle MRI scan, right? We're now seeing studies that are doing very broad MR imaging. Do you use some muscle MRI very frequently in your clinical evaluation of these patients? And if so, how? Dr Liewluck: Maybe I don't use it as much as I could, but the most common scenario I use in this setting is when I have the genetic testing come back with the VUS. So, we look at each VUS, each gene in detail. And if anything is suspicious, what I do typically go back to the literature to see if that gene defect in particular has any common pattern of muscle involvement on the MRI. And if there is, I use MRI as one of the two to try to see if I can escalate the pathogenicity of that VUS. Dr Smith: And a VUS is a “Variant of Unknown Significance,” for our listeners. I'm proud that I remember that as a geneticist. These are exciting times in neurology in general, but particularly in an inherited muscle disease. And we're seeing a lot of therapeutic development, a lot going on in Duchenne now. What's the latest in terms of disease-modifying therapeutics and gene therapies in LGMD? Dr Liewluck: Yes. So, there are several precritical and early-phase critical trials for gene therapy for the common lymphoma of muscular dystrophies. For example, the sarcoglycanopathies, and they also have some biochemical therapy that arepossible for the LGMD to FKRP. But there are many things that I expect probably will come into the picture broader or later phase of critical tryouts, and hopefully we have something to offer for the patients similar to patients with Duchenne muscular dystrophy. Dr Smith: What haven't we talked about, I mean, holy cow? There's so much in your article. What's one thing we haven't talked about that our listeners need to hear? Dr Liewluck: Good questions. So, I think we covered all, but often we get patients with proximal weakness and high CK, and they all got labeled as having limb-girdlemuscular dystrophy. What I want to stress is that proximal weakness and high CK is a common feature for muscle diseases, so they need to think broad, need to think about all possibilities. Particularly don't want to miss something treatable. Chronic, slowly progressive cause, as I mentioned earlier, we think more about muscle dystrophy, but at the cranial range, we know that rare patients with necrotic autonomyopathy and present with limb good of weakness at a slowly progressive cost. So, make sure you think about these two when suspecting that LGMD patient diabetic testing has come back inconclusive. Dr Smith: Well, that's very helpful. And fortunately, there's several other articles in this issue of Continuum that help people think through this issue more broadly. Teerin, you certainly don't disappoint. I enjoyed listening to you about a month ago, and I enjoyed reading your article a great deal and enjoy talking to you even more. Thank you very much. Dr Liewluck: Thank you very much, Gordon. Dr Smith: Again, today I've been interviewing Dr Teerin Liewluck about his article on limb-girdle muscular dystrophy, which appears in the October 2025 Continuum issue on muscle and neuromuscular junction disorders. Please be sure to check out Continuum Audio episodes for this and other issues. And thanks to our listeners for joining today. Dr Monteith: This is Dr Teshamae Monteith, Associate Editor of Continuum Audio. If you've enjoyed this episode, you'll love the journal, which is full of in-depth and clinically relevant information important for neurology practitioners. Use the link in the episode notes to learn more and subscribe. AAN members, you can get CME for listening to this interview by completing the evaluation at continpub.com/audioCME. Thank you for listening to Continuum Audio.

The FDA made waves in early September when it announced a plan to publicly release complete response letters (CRLs) at the same time they are issued to sponsoring companies, part of what the agency calls a push toward “radical transparency.” At the same time, the FDA released 89 new CRLs, adding to the more than 200 that were previously made public in July and shedding more light on the agency’s often opaque review process. That earlier document dump, though, only included rejection letters for drugs that were eventually approved. Now, for the first time, the agency is releasing CRLs for unapproved drug candidates—including recent high-profile rejections such as Ultragenyx’s gene therapy for Sanfilippo syndrome type A, Capricor Therapeutics’ Duchenne muscular dystrophy cell therapy and Replimune’s melanoma drug. Publicly airing the rationale for rejecting drug candidates is a significant shift for the FDA and a potential cause for concern for the biopharma industry. To unpack what the new policy means for drug developers, Fierce Biotech’s Darren Incorvaia sat down with Torrey Cope, a lawyer with Sidley Austin who represents and advises life sciences companies on issues related to the FDA. To learn more about the topics in this episode: FDA releases ‘initial batch’ of more than 200 drug rejection letters FDA moves to publish drug rejection letters in real time, releases new batch including Replimune's high-profile snub FDA misinterpreted efficacy data that prompted DMD rejection, Capricor claims ‘Weak’ evidence and an ‘unpleasant’ odor: FDA sheds light on drug refusal process 'Several concerning observations': FDA sheds more light on reasons it rejected drugs See omnystudio.com/listener for privacy information.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Let's dive into the latest news shaping this dynamic industry.Bristol Myers Squibb recently made headlines with their acquisition of Orbital Therapeutics for a remarkable $1.5 billion. This strategic move is aimed at enhancing their in vivo cell therapy capabilities, particularly in treating autoimmune disorders. In vivo cell therapy is a pioneering approach that allows genetic modifications directly within a patient's body, potentially revolutionizing the treatment landscape for numerous conditions. This acquisition underscores Bristol Myers Squibb's commitment to pushing the boundaries of innovative cell therapy technologies and reflects a broader trend in the industry towards personalized medicine.In another significant development, AstraZeneca has aligned with the Trump administration's Most Favored Nation pricing program, agreeing to provide Medicaid drugs at prices competitive on a global scale. This decision marks a strategic shift towards cost reduction, especially in chronic disease management and respiratory therapeutics. The move is indicative of AstraZeneca's efforts to adapt to regulatory pressures and evolving policies that emphasize value-based healthcare delivery.Meanwhile, Ypsomed has announced plans to invest $248 million in establishing a manufacturing facility in North Carolina. This facility will focus on producing auto-injectors, essential for treating diabetes and metabolic disorders. The investment signifies a strategic operational expansion aimed at meeting rising demand in North America, highlighting the growing importance of drug delivery devices in the therapeutic landscape.Turning to clinical trials, Regeneron has unveiled promising Phase 1/2 data for its DB-OTO gene therapy targeting genetic hearing loss in children. By using AAV vectors to address DFNB9-related synaptic transmission deficits, this therapy could mark a breakthrough for those suffering from hereditary hearing conditions. Satellos has also presented encouraging Phase 1 results for SAT-3247, an oral small molecule targeting AAK1 in Duchenne muscular dystrophy, with plans to proceed to Phase 2 trials focused on muscle regeneration.In oncology, Taiho and Cullinan's Phase 2 data on zipalertinib showed efficacy against EGFR-mutated non-small cell lung cancer with brain metastases. This advancement highlights the potential of tyrosine kinase inhibitors in precision oncology. Similarly, Arcus Biosciences reported a median survival of 26.7 months for its combination therapy with domvanalimab and zimberelimab in gastroesophageal adenocarcinoma trials, underscoring the promise of TIGIT-targeted therapies.Assembly Biosciences has shared promising Phase 1b results for its ABI-5366 helicase-primase inhibitor, achieving an impressive 94% reduction in herpes simplex virus shedding. OS Therapies reported significant survival improvement with its OST-HER2 vaccine in recurrent pulmonary metastatic osteosarcoma patients, positioning HER2-targeting immunotherapies as promising cancer treatment interventions.Cabaletta Bio has made strides with its resecabtagene autoleucel CAR-T therapy, demonstrating B cell elimination without preconditioning in pemphigus vulgaris trials. This innovation opens new doors for autoimmune disease management through advanced cell therapies.On the business development front, Roche's out-licensing of its GLP-1/GIP agonist CT-388 to Chugai for diabetes and obesity treatment exemplifies strategic partnerships focused on addressing metabolic disorders through novel small molecules.The sector is also witnessing significant financial activities with Evommune filing an IPO to advance treatments for inflammatory conditions. Meanwhile, Quoin Pharmaceuticals raised $104.5 million through private placement to concentrate on rare disSupport the show

Ce 6 octobre marque la Journée nationale des aidants. Près d'un adulte sur trois en France soutient au quotidien un proche en perte d'autonomie ou en situation de handicap. Si toutes les classes d'âges sont concernées, ce sont en grande majorité des personnes entre 55 et 64 ans qui sont aidantes. Et parmi elles, une très grande majorité de salariés qui, pour faire face, mettent à mal leur propre santé, jusqu'à épuisement. Il existe des aides, mais elles sont insuffisamment connues et pas assez généralisées. Notre correspondante est allée à la rencontre de Jean-Louis, proche aidant de son fils Jacques, en situation de handicap.  Un peu décoiffé, le regard volontaire, Jacques patiente dans son lit. Il est atteint de la myopathie de Duchenne, une maladie dégénérative qui atrophie les muscles. Jean-Louis, son père, est son proche aidant : « Je lève Jacques de son lit pour le mettre sur son fauteuil, c'est ce que l'on appelle un transfert. » À 22 ans et en fauteuil roulant, Jacques a besoin d'être porté au moins six fois par jour. Mais l'aide ne s'arrête pas là. « Tout ce qui est habillage, toilette, tout ce qui est brossage de dents. Je perds la force, d'année en année. Il faut m'aider à manger, même à boire. J'ai besoin d'aide pour toutes les choses dont un humain a besoin, toutes les choses quotidiennes. », explique Jacques.  Et le jeune homme se culpabilise : « Quand je l'appelle pour me tourner la nuit, et parfois, j'ai envie de le laisser dormir... », regrette Jacques. Jean-Louis regarde tendrement son fils, mais parfois cette aide constante l'épuise, il avoue : « J'ai déjà craqué. En plus, je suis assez direct. J'ai déjà dit à Jacques que je n'en pouvais plus. Cela n'est pas arrivé 36 fois, mais il y a des moments où il y a des grands coups de fatigue. Surtout lors des réveils nocturnes, quand il me réveille cinq fois dans la nuit parce qu'il a mal à la jambe. Là, je suis un peu au bout du rouleau. » À lire aussiLes aidants : 9,3 millions de Français concernés Jacques est étudiant à la fac et Jean-Louis est salarié. Ça fait dix ans que ce parent aidant consacre un nombre incalculable d'heures à son fils handicapé, mais il ne bénéficie que de quatre jours de congés supplémentaires par an. Jean-Louis aurait la possibilité d'être relayé par un professionnel, mais il regrette qu'il ne soit pas suffisamment formé : « N'est pas un bon aidant qui veut, et c'est vrai que - Jacques pourra mieux en parler -  on a des aidants qui quelques fois ne font pas grand-chose. C'est se mettre à disposition de l'autre et ne pas avoir peur : tu as besoin de quoi ? Est-ce que tu as faim ? Est-ce que tu as soif ? Tu es bien comme tu es ? Tu veux te lever ? Il y a des besoins, il y a peut-être aussi des envies : penser à ouvrir la fenêtre, le soleil va rentrer… » « Il faut qu'il soit dans la compréhension de l'autre. Il faut par exemple une formation psychologique, sur comment aider le malade », ajoute son fils.  Pour Jacques, son père est le meilleur des aidants. « Il n'est pas dans la mièvrerie. Il est dans le combat », affirme-t-il. Et ce qui est touchant, c'est que père et fils se battent ensemble. À lire aussiMieux comprendre les difficultés des aidants

Ravinder Taylor is a renowned brain training expert, author, and personal development coach who champions self-empowerment through science-based mind training techniques. Drawing from her own experiences growing up in England and overcoming insecurities, Ravinder has dedicated her career to helping others realize their true potential. Her latest book, Mind Training: The Science of Self Empowerment, distills insights from over 200 independent research studies and offers practical tools for cultivating positive change. Ravinder's approach blends research-backed evidence with actionable tips, from diaphragmatic breathing and smiling to innovative subliminal programs, making her guidance accessible and transformative for audiences everywhere.  Takeaways: You are capable of more than you think—not just physically, but mentally and emotionally—and self-belief is the foundation for growth. Simple, science-backed habits like genuine smiling and diaphragmatic breathing can immediately influence your mood, health, and longevity. Our subconscious beliefs often hold us back, but tools like subliminal messaging and intentional self-talk can help rewire those patterns for empowerment. Sound Bites: “There's always something good you can learn from even the toughest situations.” “A Duchenne smile, that big, genuine smile, can literally add years to your life.” “When you don't believe in yourself, you put a cap on everything you can possibly achieve.” Connect & Discover Ravinder: Website:  https://www.ravindertaylor.com/ Facebook: https://www.facebook.com/RavinderKTaylor Instagram: https://www.instagram.com/ravindertaylor/ Book: Mind Training:the Science of Self Empowerment

Dr. Neil Toupin, a child neurologist at UK HealthCare, shares valuable information about Duchenne muscular dystrophy for referring providers.

A global shift may be underway in biopharma as the White House prepares legislation that would would place restrictions on drugs brought to the U.S. from China, and pharma companies exit the U.K. in droves. President Donald Trump is reportedly writing an executive order that would clamp down on the pharmaceutical industry's ability to buy new molecules from biotechs based in China, while Sanofi, Merck and more have canceled or suspended investments in the U.K. following a sizeable increase in a mandatory levy in the region. In other business news, Novo Nordisk's newly appointed CEO Maziar Mike Doustdar hit the ground running, cutting around 9,000 employees and informing those who remained that they would need to return to the office. Novo's headcount had climbed 81% in five years as its revenue soared—and then fell. The obesity juggernaut has been a key presence at the European Association for the Study of Diabetes' annual meeting this week, announcing that it would seek FDA approval for a high-dose formulation of Wegovy, and presenting new data for long-acting amylin analog cagrilintide. Meanwhile, Reuters reported that Eli Lilly's orforglipron could potentially qualify for the FDA's recently launched Commissioner's Priority Voucher, which could see the oral obesity candidate approved this year.  The gene therapy space was hit with more bad news as Capsida Biotherapeutics reported the death of a patient being treated with its investigational gene therapy for epileptic disorders. This follows an unfortunate trend in 2025 that has also seen deaths attributed to Sarepta's approved Duchenne muscular dystrophy gene therapy Elevidys and a monoclonal antibody used for lymphodepletion in a study of Allogene's CAR T cell therapy cema-cell. According to a new analyst survey, however, doctors are still prescribing Elevidys to ambulatory patients.  Meanwhile, on the regulatory front, FDA Center for Drug Evaluation and Research Director George Tidmarsh reportedly told two separate groups last week that he “would like to get away” from advisory committee meetings, but quickly appeared to walk the comments back in a statement to Endpoints News.  Finally, in BioPharm Executive, BioSpace takes a deep dive into the FDA's new crackdown on pharmaceutical drug ads, and spotlights Akeso CEO Michelle Xia who built the biotech from a $3 million angel fundraising round to its current $15 billion valuation.  

Host Matt chats with American genetic counsellor Dr Lisa Schwartz about the evolving professional identity of genetic counsellors, the role of networking and qualitative research (including authors' positionality), and how clinical training translates into lab, policy and education roles. They also swap stories about travel, outdoor winery concerts, Stevie Nicks, their dogs, and advances in genetics such as Duchenne research, while discussing how training and definitions may need to change as the profession grows.

Uma campanha beneficente, lançada pela marca Nicole Barretta Acessórios, une a conscientização do Outubro Rosa a uma causa emergencial na região: a arrecadação de fundos para o tratamento de Dudu, um menino de 11 anos, de Erechim, diagnosticado com distrofia muscular de Duchenne. A iniciativa consiste na venda de um bottom (broche) temático, ao preço de R$ 19,00, com parte da renda revertida para a campanha "Salve o Dudu", que busca levantar R$ 17 milhões para uma medicação de alto custo. A ação foi detalhada pela designer de moda e proprietária da marca, Nicole Barretta, e pela revendedora Tatieli Fochesatto, em entrevista à Rádio Sideral na quarta-feira, 10 de setembro.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.A new data release shows that Summit's Keytruda competitor, Ivonescimab, had better overall survival rates in Asian patients compared to those in North America and Europe. Despite this, the drug narrowly missed the statistical significance threshold required by the FDA for approval in Western countries. Meanwhile, Rapport Therapeutics saw a 180% increase in its stock value after positive mid-stage data for its epilepsy pill, and the FDA opted for a consumer "green list" instead of addressing compounded versions of obesity drugs. The FDA's leadership turnover has led to confusion and uncertainty for small and medium biopharma companies. Lilly is seeking individuals who are willing to challenge the status quo in medicine.FDA expert panels are being criticized for lack of balance, with some meetings being one-sided. RFK Jr. faced bipartisan criticism for his anti-vaccine views during a Senate hearing. Meanwhile, Servier committed up to $530 million in an eye cancer deal and Gilead partnered with the US State Department to distribute a drug to low-income countries. The FDA released a new rare disease approval framework and dropped heavily redacted rejection letters. Amidst uncertainty in FDA leadership, small and medium biopharma companies are left without clear guidance for their therapies. Opinion pieces highlight the potential of small molecules in gene therapy for Duchenne muscular dystrophy and criticize the lack of balance in FDA expert panels. The newsletter also includes upcoming events, job listings, and a request for feedback on coverage topics.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "Overviewing Consensus Guidelines to Standardize Gene Therapy Care in Duchenne," Barry Byrne, MD, chief medical advisor at the Muscular Dystrophy Association and director of the Powell Gene Therapy Center at the University of Florida, discusses newly published consensus guidelines from the MDA and Parent Project Muscular Dystrophy on the safe delivery and monitoring of gene therapy in Duchenne muscular dystrophy (DMD). Byrne explains the rationale behind convening a global panel of experts, emphasizing the need for standardized practices in patient selection, administration, and follow-up care as gene therapy becomes more widely available. He highlights the importance of expanded multidisciplinary teams—including hematology, cardiology, nephrology, and immunology—in managing immune-related safety concerns, with particular attention to monitoring liver inflammation and emerging strategies such as rapamycin use. In addition, Byrne outlines how these guidelines address real-world challenges around access, including geographic barriers, language considerations, and financial constraints, while underscoring their role in shaping future gene therapy approaches as additional therapies move through development. Looking for more Neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page. Episode Breakdown: 1:00 – Why consensus guidelines were needed for gene therapy in DMD 2:10 – Top-line clinical considerations from the published recommendations 4:30 – Protocols for monitoring and managing adverse events, especially liver toxicity 6:30 – Neurology News Network 8:30 – Addressing health equity, language access, and financial barriers in gene therapy care 12:00 – How these recommendations may shape the future of DMD treatment The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Efgartigimod Aims to Become First Therapy for Seronegative Generalized Myasthenia Gravis Following Positive Phase 3 Data FDA Approves Lecanemab Autoinjector, Marking First At-Home Treatment for Alzheimer Disease Eisai Submits sBLA for Weekly Subcutaneous Lecanemab as Starting Dose Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Conversamos con Ángeles Pérez, madre de Luis, un joven que sufre el síndrome de Duchenne

Elijah Stacy, founder of Destroy Duchenne, speaks with Kevin Counterman, author of Breakfall, along with Buddy and Alan, about their new book. Breakfall is inspired by Kevin's late brother, Michael, and tells the story of Rudy and the Gallagher family, exploring themes of legacy, resilience, and the impact of Duchenne muscular dystrophy on the whole family.Learn More about Breakfall: https://breakfallbook.comWant to be on the podcast? Click here: https://www.destroyduchenne.org/podca…Learn more about the Destroy Duchenne Podcast here: https://www.destroyduchenne.org/podcastWebsite: https://www.destroyduchenne.orgSocial Media: Facebook:   / destroyduche.  .Instagram:   / destroyduch.  .Twitter: https://twitter.com/destroydisease?la...LinkedIn:   / dest.  .ABOUT DESTROY DUCHENNE PODCAST:A podcast telling the stories of those affected by a fatal muscle-wasting disease, Duchenne muscular dystrophy (DMD), that robs children of their ability to walk by the age of 11, the ability to raise their arms by their teenage years, and eventually their lives in their early 20s.Hosted by 24-year-old founder of the 501(c)3 nonprofit organization Destroy Duchenne, Elijah Stacy, who also suffers from Duchenne muscular dystrophy.The goal of the Destroy Duchenne podcast is very simple, yet ambitious: interview every person that has a relationship to Duchenne—people with Duchenne, parents, family members, friends, teachers, doctors—and tell their story to the world.Destroy Duchenne is a registered 501(c)3 nonprofit organization with the mission: to Complete the Cure for Duchenne muscular dystrophy by advancing medical technologies into human practice.Federal Tax ID: #82-3268952#Breakfall

Sunnudagurinn næsti verður Duchenne dagurinn, en Duchenne er sjaldgæfur vöðvasjúkdómur sem skerðir hreyfigetu og hrjáir einn af hverjum fjögur þúsund drengjum. Við fræddumst um nýja íslenska heimildarmynd sem heitir Einstakt ferðalag. Hún er um Ægi Þór 9 ára dreng frá Höfn í Hornafirði, sem er með þennan sjaldgæfa sjúkdóm, og ferðalag hans um Ísland þar sem hann hittir önnur börn með sjaldgæfa sjúkdóma. Myndin varpar ljósi á stöðu langveikra barna og aðstandenda þeirra hér á landi. Ágústa Fanney Snorradóttir, leikstjóri myndarinnar, kom í þáttinn og sagði okkur betur frá henni. Jóhanna Birna Bjartmarsdóttir hefur á síðustu misserum vakið athygli en hún hélt athyglisverðan fyrirlestur á ráðstefnu BUGL árið 2023 þar sem hún fór yfir reynslu sína af íslenska skólakerfinu og hvernig henni fannst það bregðast sér. Jóhanna Birna er greind með lesblindu, einhverfu og ADHD. Jóhanna Birna hefur nú lokið háskólanámi í Bandaríkjunum og hyggur á áframhaldandi nám. Hún heldur fyrirlestra um hvernig henni finnst að skólakerfið geti betuð komið til móts við börn í svipuðum aðstæðum og hún var í þegar hún gekk í grunnskóla. Svo kom Einar Sveinbjörnsson veðurfræðingur til okkar í veðurspjallið í dag. Hann var með uppgjör á sumarveðrinu og hverrnig við upplifum það á mismunandihátt. Einar talaði svo um hitamet og metsumur úti í heimi, horfurnar næstu daga og að lokum reyndi Einar að svara spurningunni: Hvenær mun hausta? Tónlist í þættinum í dag: Heiðlóan / Gísli Magna og Co. (Steingrímur M. Sigfússon) Þótt falli snjór / Jóhann Sigurðason (Ágúst Guðmundsson) Landleguvalsinn / Haukur Morthens (Jónatan Ólafsson, texti Númi Þorbergsson) Lítið og væmið / Valdimar (Valdimar Guðmundsson) UMSJÓN: GUNNAR HANSSON OG GUÐRÚN GUNNARSDÓTTIR

2025-08-22 Hosts Craig Lipset and Jane Myles hosted a conversation with Alpa Kushillani, VP of Customer Success & Delivery with Gooseberry Research and a rare disease advocate. Alpa shares her family's journey with Duchenne muscular dystrophy and how those experiences inspired her to create new solutions for patient-centered research.We explore the realities of clinical trial participation for rare disease families—from challenges in home healthcare and nurse training to the promise of digital health tools and decentralized approaches. Alpa highlights how Gooseberry Research is working to improve trial accessibility through specialized nurse training, stronger site communication, and partnerships with technology providers.The episode also touches on trial design hurdles, the need for meaningful endpoints, and the future of decentralized clinical trials. Episode Resources:DCT Market TrendResearch About a Novel, Home-Based Assessment for Duchenne Muscular DystrophyYou can join TGIF-DTRA Sessions live on LinkedIn Live on Friday's at 12:00 PM ET by checking out our LinkedIn. Follow the Decentralized Trials & Research Alliance (DTRA) on LinkedIn and X. Learn more about Membership options and our work at www.dtra.org.

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 

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Sarepta has released new safety data for their gene therapy Elevidys, showing zero ambulatory deaths in patients with Duchenne muscular dystrophy. This data confirms the therapy's positive risk/benefit profile. Meanwhile, Novo Nordisk's rapid rise and fall was driven by GLP-1s like Ozempic and Wegovy, leading to a plummet in stock value. Insmed has received FDA approval for the first bronchiectasis drug and DPP1 blocker, Brinsupri. Texas has accused Eli Lilly of "bribing" prescribers to push their drugs Mounjaro and Zepbound, leading to a new lawsuit. Genscript is entering a new era of innovation and trust, offering exclusive promos and events. Additionally, Tang Capital is on a buyout binge in the biotech industry, while a top ARPA-H official has departed in protest of cuts to mRNA funding. The biotech industry continues to see contractions with companies like AbSci, Fate, and Oric downsizing. In career advice, loyalty is seen as a flawed strategy that can cost individuals opportunities.Arrowhead, a biotech company, has emerged as a savior in the RNA interference (RNAi) pipeline after Sarepta faced troubles unrelated to Arrowhead's assets. Concentra Biosciences, a biotech shell company, has been on a buying spree, acquiring four biotechs in the past month and seven so far this year. President Trump's most favored nation drug pricing policy is seen as unfavorable for all parties involved. Novo Nordisk experienced a rapid rise and fall due to the success of ozempic and wegovy. Lilly is facing drug pricing pressure while defending its injectable GLP-1 empire. Novartis is rumored to be considering acquiring RNA specialist Avidity. Despite safety concerns, Sarepta beat Q2 estimates, but not due to sales of its product Elevidys. Various biotech companies are downsizing while others are receiving significant investments.

Vinay Prasad is back at the FDA as chief of the FDA's Center for Biologics Evaluation and Research. Prasad's return—which hit the news wires Saturday morning—came just 10 days after his unexpected exit on July 29, following blowback over the saga involving Sarepta Therapeutics' Duchenne muscular dystrophy gene therapy Elevidys, and a campaign by conversative personalities to oust the outspoken physician.Elsewhere in the government, Gray Delany, former head of HHS' and President Donald Trump's Make America Healthy Again agenda, has been fired—days after Health and Human Services Secretary Robert F. Kennedy Jr. axed 22 mRNA vaccine contracts under the Biomedical Advanced Research and Development Authority. Delany's ouster came after he allegedly butted heads with other agency officials over how announcements were made, according to reporting from Endpoints News. The mRNA contract cuts, meanwhile, have sparked criticism from the scientific community and concern that the growing anti-mRNA sentiment could also have a negative effect on research for cancer therapeutics.In somewhat related news, the Annals of Internal Medicine is refusing to retract a large-scale study it ran in July that pointed to the lack of an association between childhood aluminum exposure through vaccination and chronic conditions, despite Kennedy's criticism. In an op-ed published earlier this month, the Health Secretary took issue with the study's design.In obesity news, biopharma darling Eli Lilly suffered a rare chink in its normally impenetrable GLP-1 armor. The first Phase III trial for oral weight loss therapy orforglipron read out last week, and the results were underwhelming, at least to Wall Street. Truist Securities wrote that the data support approval but “leaves room for competition.” This is good news for a whole host of companies, including Roche, Viking, Terns and Rhythm Pharmaceuticals, who all have weight loss pills at various stages of development.And in gene therapy, the hits just keep coming. The FDA has limited the use of bluebird bio's gene therapy Skysona to patients with cerebral adrenoleukodystrophy for whom no other therapies or stem cell donors are available, due to a heightened risk of blood cancers. This follows safety issues in gene therapy trials experienced by Allogene, and, of course, Sarepta Therapeutics. This week in ClinicaSpace, Dan Samorodnitsky explored the future of AAV technology. And in BioPharm Executive, Annalee Armstrong sat down with Chris Anzalone, CEO of Arrowhead Pharmaceuticals—Sarepta's key strategic partner—to learn how his company has been weathering the storm.

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

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.

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.

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.

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

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.”

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            

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.

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.