Zolgensma Clinical Trials and Their Results
Zolgensma Clinical Trials and Their Results
The first in-human trial for intravenous (IV) administration of Zolgensma STRIVE began in April 2014. This and the Phase 1 START follow-up trial led to the eventual approval of this one time gene therapy in both the US and Europe for children with SMA Type 1.
In April 2021, a new global clinical trial investigating the safety, tolerability and efficacy of intravenous Zolgensma™ in children (other than those diagnosed with SMA Type 1) who have SMA and weigh between 8.5 kgs and 21 kgs.
In August 2021, the clinical trial of intrathecally (IT) administered Zolgensma resumed and was called STEER. It reported in January 2025.
Results and Updates for Each Trial >