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Risdiplam in adult non-sitter patients with 5q spinal muscular atrophy: a non-interventional, single centre, observational cohort study

2022-06-21 neurology Title + abstract only
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ObjectiveTo describe the safety and efficacy of risdiplam in non-sitter adult patients with 5q spinal muscular atrophy (SMA). MethodsType 2 SMA adult patients, who were not eligible for nusinersen, were offered risdiplam through the expanded access program. Patients were followed up with a battery of scales and clinical measures. ResultsSix non-sitter patients (17 - 46 years old) were treated with risdiplam. One patient reported mild adverse events (dyspepsia and headache). After one year of t...

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