Special Episode: SRP-9001 Approved As First Gene Therapy for Duchenne Muscular Dystrophy

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of SRP-9001, or delandistrogene moxeparvovec (Elevidys; Sarepta) in Duchenne muscular dystrophy (DMD). Offering commentary is Natalie Goedeker, CPNP, a nurse practitioner in neurology in the Neuromuscular Division at Washington University in St Louis. SRP-9001 is an AAV vector-based gene therapy that was approved for the treatment of ambulatory patients with DMD with a confirmed mutation in the DMD gene aged 4 to 5 years, based on data from the phase 3 EMBARK study (NCT05096221). For more of NeurologyLive®'s coverage of SRP-9001's approval, head here: FDA Approves SRP-9001 as First Gene Therapy for Duchenne Muscular Dystrophy Episode Breakdown: 0:30 – SRP-9001 (Elevidys; Sarepta) is approved for DMD 1:35 – Natalie Goedeker, CPNP, on the approval 3:05 – Efficacy of SRP-9001 4:00 – Goedeker on pieces of efficacy data released 4:45 – Currently available therapies for DMD 5:35 – Goedeker on role of SRP-9001 in treatment landscape 6:55 – Safety data of SRP-9001 8:00 – Goedeker on safety considerations of which prescribing clinicians should be aware 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. REFERENCES 1. Sarepta Therapeutics announces FDA approval of Elevidys, the first gene therapy to treat Duchenne muscular dystrophy. News release. June 22, 2023. Accessed June 22, 2023. https://www.businesswire.com/news/home/20230622454844/en/