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Shifting the Treatment Paradigm in Spinal Muscular Atrophy


Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease.

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.

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