Publications & Posters
Scholarly publications
Spinal Muscular Atrophy
Apitegromab
October 2025
World Muscle Society Annual Congress
Pharmacokinetics (PK) and pharmacodynamics (PD) of apitegromab in spinal muscular atrophy
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 (poster)
Spinal Muscular Atrophy
Apitegromab
September 2025
Neuromuscular Study Group Annual Scientific Meeting
Characterization of the longer-term effectiveness of SMN-targeted treatments for spinal muscular atrophy: A systematic literature review
Spinal Muscular Atrophy
Apitegromab
September 2025
Neuromuscular Study Group Annual Scientific Meeting
