New Phase 3 Data Show Success for Treating Spinal Muscular Atrophy with Gene Therapy


New data demonstrates that onasemnogene abeparvovec is an effective treatment option for symptomatic and presymptomatic patients with spinal muscular atrophy (SMA).

Jerry Mendell, MD

Jerry Mendell, MD

Data from 2 long-term studies demonstrated the sustained efficacy and durability of onasemnogene abeparvovec (Zolgensma) in children, a one-time gene therapy for the treatment of spinal muscular atrophy (SMA). The studies, LT-001 and LT-002, showed that the therapy had a favorable benefit-risk profile across a range of patient populations.1

The ongoing LT-001 study, which followed patients who completed the phase 1 START study for 15 years, showed that children who were treated after presenting symptoms of spinal muscular atrophy maintained all previously achieved motor milestones up to 7.5 years post-dosing. Additionally, 3 patients achieved the ability to stand with assistance during the study period.

“I have had the privilege of observing some of the children included in the (long-term follow-up) studies since they started their Zolgensma clinical trial journey, and the fact that we’re seeing them maintain and, in some cases, gain motor milestones when they are nearly 8 years old is truly transformational,” Jerry Mendell, MD, Nationwide Children’s Hospital said in a statement.

In the LT-002 study, 100% of patients maintained motor milestones, including both presymptomatic and symptomatic populations. This success was observed in those treated via intravenous (IV) and intrathecal (IT) administration methods, with 100% of patients maintaining motor milestones achieved during their respective parent studies in the follow-up period.

These new data demonstrated that onasemnogene abeparvovec was an effective and durable treatment option for symptomatic and presymptomatic patient populations.

“These children now have an improved quality of life, vastly different from what would have been expected for them if they had not received treatment,” Mendell stated. “I am excited to see the new possibilities that open up to the children, their families and others who may now be able to receive this treatment.”


  1. 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. News release. Navartis Therapeutics. March 20, 2023. Accessed March 20, 2023.
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