Strategic Insights Into Evolving Gene Therapy Trends

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

EP: 10.Duchenne Muscular Dystrophy Treatment Landscape Review

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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 Ciafaloni and Nance, focusing on advancements in gene therapy and early screening for genetic diseases. Ciafaloni emphasizes the importance of long-term data to demonstrate clinical benefits and discusses innovative gene-based therapies like exon skipping and peptide-conjugated phosphorodiamidate morpholino oligomer with improved target effects and dosing intervals. Nance underscores the significance of early screening, drawing parallels with successful initiatives in spinal muscular atrophy and advocating for expanded newborn screening to facilitate timely intervention for conditions like Duchenne muscular dystrophy. The panelists explore the challenges and opportunities in integrating genetic screening technologies into health care systems, envisioning a future where early identification and targeted interventions optimize patient outcomes in genetic diseases. Haumschild concludes by emphasizing the critical role of proactive screening strategies in realizing the full potential of gene therapy and improving health care delivery for affected individuals.

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