149: Overviewing Consensus Guidelines to Standardize Gene Therapy Care in Duchenne

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.