Last Updated: 09/26/2026
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Found 32 publications
Advancing treatment of spinal muscular atrophy through inhibition of the myostatin signaling pathway.
Journal: Expert review of neurotherapeutics
Published: January 22, 2026
Opportunities for RNA sequencing in physiology: from big data to understanding homeostasis and heterogeneity.
Journal: Function (Oxford, England)
Published: December 16, 2025
Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series.
Journal: Advances in therapy
Published: December 12, 2025
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
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial.
Journal: The Lancet. Neurology
Published: March 25, 2025
Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series.
Journal: BMC neurology
Published: January 17, 2025
Access to novel therapies for Duchenne muscular dystrophy-Insights from expert treating physicians.
Journal: Annals of the Child Neurology Society
Published: May 08, 2024
Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study.
Journal: Frontiers in neurology
Published: April 18, 2024
Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations.
Journal: Neurology. Clinical practice
Published: November 27, 2023
Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care.
Journal: Biotech (Basel (Switzerland))
Published: August 24, 2023
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.
Journal: Nature medicine
Published: February 18, 2022
Last Updated: 09/26/2026