New long-term results of Zolgensma

In a press release dated 20 March 2023, Novartis presents new results for Zolgensma (onasemnogene abeparvovec) from two 15-year follow-up studies:

  • Study LT 001 is conducted in 10 participants of the START trial who had been treated post-symptomatically with intravenous therapy before the age of 6 months: all are alive, able to feed orally and do not require permanent ventilation. Six of them are standing with assistance.
  • Study LT 002 involved participants treated intravenously (from the various post-symptomatic STR1VE trials and the pre-symptomatic SPR1NT trial) or intrathecally (from the post-symptomatic STRONG trial): in all cases, motor development was maintained or improved and all pre-symptomatically treated participants had the ability to walk independently


Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset. Novartis. Press release dated 20 March 2023.