Early Life Safety Profiling of Gene Therapy for Spinal Muscular Atrophy: A Case Series Analysis
Rebecca G. Spellman, Leillani L. Ha, Salomé Da Silva Duarte Lepez, Elizabeth A. Arruda, Emma Rodrigues, Kathryn J. Swoboda, Christiano R. R. Alves
This study evaluates the safety of onasemnogene abeparvovec gene therapy in SMA patients, showing transient liver enzyme elevations that normalized, with no impact from concurrent nusinersen treatment.
Gene Therapy · Apr 1, 2025Peer-reviewedGenetic & Genomic Medicine