Publications & Posters
Scholarly publications
Spinal Muscular Atrophy
Apitegromab
February 2026
Treat-NMD International Conference
Understanding adult patient perspectives on the clinical meaningfulness of “any point differences” on the Hammersmith Functional Motor Scale–Expanded in SMA
Spinal Muscular Atrophy
Apitegromab
February 2026
Treat-NMD International Conference
Assessing the impact of “any point differences” or “stability” in the Hammersmith Functional Motor Scale–Expanded on activities of daily living in adult patients with SMA
Spinal Muscular Atrophy
Apitegromab
January 2026
British Paediatric Neurology Association Annual Conference
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
January 2026
Congrés de la Société Française de Neurologie Pédiatrique
Conception et justification de l’étude OPAL, une étude de phase 2, randomisée et en double aveugle sur l’apitegromab chez des patients âgés de <2 ans atteints de SMA
Spinal Muscular Atrophy
Apitegromab
October 2025
Child Neurology Society Annual Meeting
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
Child Neurology Society Annual Meeting
