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Evaluating Transformative Gene Therapies


Ryan Haumschild, PharmD, MS, MBA, and Mary Pak, MD, FACP, consider FDA-approved gene therapies for hemophilia, spinal muscular atrophy, and Duchenne muscular dystrophy, exploring their genetic foundations, the FDA approval process, and considerations for therapeutic value and cost.

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.

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