Article

Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the three first years of treatment.

2023-09-09

Abstract excerpt

<title>Abstract</title> <p><bold>Background</bold>: Spinal muscular atrophy type 1 (SMA1) is the most severe and early form of SMA, a genetic disease with motor neuron degeneration. Onasemnogene abeparvovec gene therapy (GT) has changed SMA1’s natural history, but real-world data is scarce. <bold>Methods</bold>: A French national expert committee identified 95 newly diagnosed and treatment-naive SMA1 cases betwee...

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Literature Corpus work: 23b0f3bf-e986-5315-89e6-4824403b641e
DOI: 10.21203/rs.3.rs-2876020/v1

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