Abstract
IV onasemnogene abeparvovec (formerly AVXS-101) is a one-time gene therapy designed to deliver a fully functional copy of the human survival motor neuron (SMN) gene to address the genetic root cause of SMA. This study assesses the safety of IV onasemnogene abeparvovec in SMA patients (pts) across all clinical investigations and includes 4 clinical trials (START, NCT02122952; STR1VE-EU, NCT03461289; STR1VE-US, NCT03306277; SPR1NT, NCT03505099), the United States Managed Access Program, the long-term registry (RESTORE), and post-marketing data.
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