Summary: 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.

Summary: Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis.

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

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

Summary: Evolving Surgical Management for Early-Onset Scoliosis in Spinal Muscular Atrophy Type 1 Given Improvements in Survival.

Summary: Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.

Summary: Risdiplam in Type 1 Spinal Muscular Atrophy.

Summary: Stress induced ketoacidosis in spinal muscular atrophy. Report of one case.

Summary: Two Years of Improved Neurological Function With Nusinersen in a 48-Year-Old Patient With Spinal Muscular Atrophy Type 3.

Summary: Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study.

Summary: Early Results of a Management Algorithm for Collapsing Spine Deformity in Young Children (Below 10-Year Old) With Spinal Muscular Atrophy Type II.

Summary: Treatment expectations and patient-reported outcomes of nusinersen therapy in adult spinal muscular atrophy.