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

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14d ago 15:31

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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

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