Duchenne Muscular Dystrophy Treatment Landscape Review

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

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

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

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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 moderates a discussion with Pak, who provides an insightful comparison of treatment options for Duchenne muscular dystrophy (DMD). Pak highlights the potential benefits of gene therapy in preserving muscle function and slowing disease progression, contrasting it with the limitations and adverse effects of steroid-based therapies. She underscores the importance of early intervention and holistic care in managing DMD, stressing the need for vigilant monitoring and timely intervention to address complications like respiratory and cardiac issues. Additionally, Pak discusses emerging challenges in gene therapy, including distribution efficiency and high treatment costs, prompting a conversation about health care decision-making and the imperative of collaborative efforts to ensure optimal patient outcomes while navigating cost constraints. Haumschild and the panelists delve into the complexities of treatment selection, inclusion criteria, and cost containment strategies, underscoring the critical role of multidisciplinary care teams in advancing DMD management paradigms.

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