Shifting the Treatment Paradigm in Spinal Muscular Atrophy

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

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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 leads a discussion with Ciafaloni on the approval of onasemnogene abeparvovec-xioi, a gene therapy for spinal muscular atrophy (SMA), in 2019. Ciafaloni reflects on the transformative impact of this treatment, marking a significant breakthrough after decades of research. Onasemnogene abeparvovec-xioi offers hope for infants with SMA, who previously faced limited treatment options and a bleak prognosis. Ciafaloni highlights the remarkable shift in SMA treatment paradigms, emphasizing the unprecedented clinical efficacy and the opportunity for early intervention through newborn screening. The conversation underscores the broader implications of onasemnogene abeparvovec-xioi’s approval, inspiring advancements in gene therapy across various disease states. Haumschild acknowledges the profound hope onasemnogene abeparvovec-xioi brings to researchers and clinicians, signaling a new era of possibility for rare disease therapies. The dialogue recognizes the collaborative efforts that led to onasemnogene abeparvovec-xioi’s approval and underscores its potential to pave the way for innovative treatments in other disease areas.

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