Autori: Darras BT

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

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

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

Risdiplam in Type 1 Spinal Muscular Atrophy

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

2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants

Revised upper limb module in type II and III spinal muscular atrophy: 24-month changes

Different trajectories in upper limb and gross motor function in spinal muscular atrophy

Age related treatment effect in type II Spinal Muscular Atrophy pediatric patients treated with nusinersen

Nusinersen in pediatric and adult patients with type III spinal muscular atrophy

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