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Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy

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To provide a greater understanding of the tolerability, safety and clinical outcomes of onasemnogene abeparvovec in real‐world practice, in a broad population of infants with spinal muscular atrophy (SMA). Click to show full abstract

To provide a greater understanding of the tolerability, safety and clinical outcomes of onasemnogene abeparvovec in real‐world practice, in a broad population of infants with spinal muscular atrophy (SMA).

Keywords: safety; muscular atrophy; abeparvovec spinal; onasemnogene abeparvovec; spinal muscular

Journal Title: Annals of Clinical and Translational Neurology
Year Published: 2022

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