Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

This is the first description of safety data for intravenous onasemnogene abeparvovec, the only approved systemically administered gene-replacement therapy for spinal muscular atrophy.We comprehensively assessed the safety of intravenous onasemnogene abeparvovec from preclinical studies, clinical studies, and postmarketing data.Single-dose toxicity studies were performed in neonatal mice and juvenile or neonatal cynomolgus nonhuman primates...