Review of Clinical Evidence for Delandistrogene Moxeparvovec for DMD

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

EP: 7.Developments in Muscular Dystrophy Gene Therapy

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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 engages Nance in a detailed exploration of the FDA’s accelerated approval of delandistrogene moxeparvovec-rokl for Duchenne muscular dystrophy (DMD) in young patients. Nance navigates the complexities of trial design for DMD, emphasizing the challenge of demonstrating therapeutic efficacy amid the disease’s slow progression. She outlines the key factors considered for approval, including safety, target engagement, and functional outcomes. Through meticulous analysis of clinical trial data, Nance reveals significant increases in dystrophin expression and decreases in creatine kinase levels post treatment, indicating a positive impact on muscle integrity. However, the interpretation of functional outcomes posed challenges, particularly in older patients, highlighting the importance of age stratification in assessing treatment efficacy. Nance’s insights underscore the nuanced considerations involved in accelerated approval pathways for gene therapies, shedding light on the intricate balance between scientific evidence and clinical outcomes in pediatric DMD treatment.

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