Publications & Posters
Scholarly publications
Spinal Muscular Atrophy
Apitegromab
October 2025
World Muscle Society Annual Congress
Trial design and rationale for OPAL, a Phase 2, randomized, double-blind study of apitegromab in patients aged <2 years with spinal muscular atrophy
Spinal Muscular Atrophy
Apitegromab
October 2025
World Muscle Society Annual Congress
Treatment effects and perceptions among apitegromab-treated patients with Type 2/3 spinal muscular atrophy: patient and caregiver interviews from the TOPAZ clinical trial
Spinal Muscular Atrophy
Apitegromab
October 2025
World Muscle Society Annual Congress
Efficacy and safety of apitegromab in patients aged 13-21 years with Type 2 or 3 spinal muscular atrophy: outcomes from the SAPPHIRE Phase 3 trial (oral presentation)
Spinal Muscular Atrophy
Apitegromab
October 2025
World Muscle Society Annual Congress
Efficacy and safety of apitegromab in patients aged 13-21 years with Type 2 or 3 spinal muscular atrophy: outcomes from the SAPPHIRE Phase 3 trial (poster)
Spinal Muscular Atrophy
Apitegromab
September 2025
Neuromuscular Study Group Annual Scientific Meeting
Efficacy and safety of apitegromab in individuals with type 2 and type 3 spinal muscular atrophy evaluated in the phase 3 SAPPHIRE trial
Spinal Muscular Atrophy
Apitegromab
September 2025
Neuromuscular Study Group Annual Scientific Meeting
