Evaluating Transformative Gene Therapies

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

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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

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 on recent gene therapies with Pak, focusing on their transformative impact on diseases like hemophilia. Pak highlights the genetic basis of hemophilia and how gene therapy addresses the underlying mutations. With FDA approval for therapies, the paradigm of hemophilia treatment shifts from prophylactic dosing to durable gene correction. The discussion underscores the potential for gene therapy to offer a 1-time cure, revolutionizing patient care and quality of life. While acknowledging cost concerns, the panel emphasizes the long-term benefits and patient advocacy perspectives. Pak emphasizes the shift from traditional treatments involving blood products to gene therapy’s targeted correction of genetic mutations, leading to a potential cure. The conversation anticipates further discussions on cost-effectiveness and access, recognizing the need for comprehensive evaluation of gene therapy’s societal and financial impacts.

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