Mechanism of action

ZOLGENSMA is a gene therapy designed to treat the genetic root cause of SMA (deletions of the SMN1 gene) by delivering a copy of a fully functioning human SMN gene1

Watch: How ZOLGENSMA works

Dosing and infusion

Preparing and infusing ZOLGENSMA

Dosing and infusion

STR1VE

A Phase 3 clinical trial

STR1VE