Developments in Muscular Dystrophy Gene Therapy

EP: 1.Overview of Gene Therapy Approaches and Impacts

EP: 2.Key Factors in Gene Vector Selection

EP: 3.Viral Vectors in Gene Therapy

EP: 4.Navigating Gene Therapy Eligibility: The Role of Genetic Testing

EP: 5.Evaluating Transformative Gene Therapies

EP: 6.Shifting the Treatment Paradigm in Spinal Muscular Atrophy

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EP: 7.Developments in Muscular Dystrophy Gene Therapy

EP: 8.Review of Clinical Evidence for Delandistrogene Moxeparvovec for DMD

EP: 9.Driving Clinical Insights: Harnessing Functional Assessments in DMD Research

EP: 10.Duchenne Muscular Dystrophy Treatment Landscape Review

EP: 11.Strategic Insights Into Evolving Gene Therapy Trends

EP: 12.Sustainable Gene Therapy Coverage Approaches

EP: 13.Interpreting Clinical Data to Guide Coverage Decisions for Rare Disease Therapies

EP: 14.Coverage of Gene Therapies: Balancing Cost, Clinical Impact, and Durability of Effect

EP: 15.Innovative Payment Models for Gene Therapies

EP: 16.Ethical Implications of Patient Access Restrictions

EP: 17.Enhancing Collaboration for Patient-Centered Care in Rare Diseases

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.