Raggruppamento Primario: Malattie Neuromuscolari

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Data

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

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

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

Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

Communicative development inventory in type 1 and presymptomatic infants with spinal muscular atrophy: a cohort study

Evolution of neuropsychological and behavioral profile in a cohort of pediatric patients with Becker muscular dystrophy in a longitudinal study

KLHL40-Related Myopathy: A Systematic Review and Insight into a Follow-up Biomarker via a New Case Report.

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