Raggruppamento Secondario: Atrofia Muscolare Spinale (SMA)

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

Patient and parent oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in SMA. Rome, 13 July 2019

First international workshop on rehabilitation management and clinical outcome measures for spinal muscular atrophy

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

A 5-center experience with intrathecal administration of nusinersen in SMA1 in Italy letter to the editor of european journal of pediatric neurology regarding the manuscript “single-center experience with intrathecal administration of nusinersen in children with spinal muscular atrophy type 1″ written by pechmann and colleagues”

Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

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

Risdiplam in Type 1 Spinal Muscular Atrophy

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