Publications & Posters
Scholarly publications
Spinal Muscular Atrophy
Apitegromab
August 2025
SMA Europe Clinical Care Symposium
Characterization of the longer-term effectiveness of SMN-targeted treatments for spinal muscular atrophy: A systematic literature review
Spinal Muscular Atrophy
Apitegromab
August 2025
The Lancet Neurology
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
Neuromuscular
Spinal Muscular Atrophy
Apitegromab
June 2025
Cure SMA Research & Clinical Care Meeting
Efficacy and Safety of Apitegromab in Individuals with Type 2 and Type 3 Spinal Muscular Atrophy Evaluated in the Phase 3 SAPPHIRE Trial
Neuromuscular
Spinal Muscular Atrophy
Apitegromab
June 2025
Cure SMA Research & Clinical Care Meeting
Characterization of the Longer-Term Effectiveness of SMN-Targeted Treatments for Spinal Muscular Atrophy: A Systematic Literature Review
Facioscapulohumeral Muscular Dystrophy
Neuromuscular
SRK-015
June 2025
FSHD Society International Research Congress
SRK-015 improves muscle mass, strength, and endurance in the FLExDUX4.Cre mouse model of FSHD
Spinal Muscular Atrophy
Apitegromab
March 2025
Muscular Dystrophy Association Clinical & Scientific Conference
