Last Updated: 09/26/2026
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Found 197 publications
Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.
Journal: The Lancet. Child & adolescent health
Published: January 28, 2026
Author Correction: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Journal: Nature medicine
Published: January 28, 2026
Advancing treatment of spinal muscular atrophy through inhibition of the myostatin signaling pathway.
Journal: Expert review of neurotherapeutics
Published: January 22, 2026
A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophy.
Journal: Expert review of neurotherapeutics
Published: November 13, 2025
Multifrequency Electrical Impedance Myography Enhanced with Machine Learning for Screening Patients with Neuromuscular Disorders.
Journal: Annals of biomedical engineering
Published: November 11, 2025
Refining functional phenotypes in an international cohort of untreated paediatric type 2 and 3 SMA patients using the Revised Hammersmith Scale.
Journal: Neuromuscular disorders : NMD
Published: October 10, 2025
Loss of ambulation in SMA III at the time of disease-modifying treatments: an international study.
Journal: Journal of neurology, neurosurgery, and psychiatry
Published: August 28, 2025
Differences in swallowing efficacy of disease modifying treatment between infants receiving pre-symptomatic and symptomatic administration.
Journal: Orphanet journal of rare diseases
Published: August 21, 2025
Addressing the Global Disparities in Access to Treatment in Spinal Muscular Atrophy.
Journal: Muscle & nerve
Published: July 29, 2025
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Journal: Nature medicine
Published: July 23, 2025
Further Comments on Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy.
Journal: The New England journal of medicine
Published: July 02, 2025
Last Updated: 09/26/2026