Standardizing Gene Therapy Care in Duchenne: An Overview of Consensus Guidelines

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Episode 149 of the NeurologyLive^® Mind Moments^®  is now live! Scroll down to listen or  to subscribe on your favorite streaming service.

The Mind Moments podcast features exclusive interviews with leaders in neurology discussing the latest research and disease management strategies across the breadth of the field, including epilepsy, multiple sclerosis, Parkinson disease (PD), dementia, sleep disorders, and more.

In this episode, "Overviewing Consensus Guidelines to Standardize Gene Therapy Care in Duchenne," Barry Byrne, MD, PhD, the chief medical advisor at the Muscular Dystrophy Association and the director of the Powell Gene Therapy Center at the University of Florida, speaks on the MDA and Parent Project Muscular Dystrophy's new consensus guidelines, which were recently published and cover safe delivery and monitoring practices for Duchenne muscular dystrophy (DMD) gene therapy. Byrne stressed the necessity of standardized practices for patient selection, administration, and follow-up care, especially as wider availability of gene therapy is dawning, which explain the rationale for bringing together a panel of experts from across the globe. Byrne also drew attention to the key role of expanded multidisciplinary teams with experts in hematology, cardiology, nephrology, and immunology, for the management of immune-related safety concerns. In particular, he pointed out that special attention need to be paid to liver inflammation monitoring and emerging strategies like the use of rapamycin. Furthermore, Byrne also went over geographic barriers, language considerations, and financial constraints—the real-world challenges surrounding access—and emphasized the role of these concerns in guiding the future of gene therapy approaches that continue to be developed.

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