Autori: Zanoteli E

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Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

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