Page last checked: 22nd November 2022

Last updated: 27th March 2020

New data from the START long-term follow-up study continues to demonstrate the durability of a single, one-time dose of Zolgensma in patients now up to five years after their treatment. Some of these Type 1 patients are now more than five years of age.

All patients in this study who received Zolgensma were alive, free of permanent ventilation and continued to maintain developmental milestones. This includes two patients who achieved the new milestone of standing with assistance during the long-term follow-up period.

Cumulative Safety Data

The safety data that has accumulated across all trials of Zolgensma from 335 patients by this time indicate that it is generally safe and well tolerated. Nearly all patients experienced adverse events, however, most were not serious and unrelated to the treatment.