Opinion

Video

Developments in Muscular Dystrophy Gene Therapy

Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the transformative potential of gene therapies.

This is a video synopsis/summary of a Peer Exchange involving Ryan Haumschild, PharmD, MS, MBA; Jessica Nance, MD, MS; Kimberly C. Chen, DO, MSHLM; Emma Ciafaloni, MD, FAAN; and Mary Pak, MD, FACP.

Haumschild leads a discussion with Ciafaloni on gene therapy for muscular dystrophies, with a focus on Duchenne muscular dystrophy (DMD). Ciafaloni highlights the complexity of DMD, emphasizing its genetic basis and role in muscle structure. Despite challenges, recent advancements have paved the way for gene therapy strategies targeting DMD. The conversation delves into the disease’s progression, outlining its impact on motor function and survival. Ciafaloni underscores the urgent need for disease-modifying treatments, given the limitations of current supportive care. Gene therapy holds promise as a transformative approach, offering restoration of dystrophin at the genetic level through exon skipping and gene replacement. The discussion explores the potential of combination therapies to enhance outcomes and improve patient care. Ciafaloni’s insights shed light on the evolving landscape of muscular dystrophy treatment, driven by rapid advancements in gene-based therapies and newborn-screening initiatives.

Video synopsis is AI-generated and reviewed by AJMC® editorial staff.

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