Results from the one-year SMART trial with Zolgensma, the Novartis gene therapy product, were announced by Novartis via press release and presented at the Muscular Dystrophy Association (MDA) annual meeting:
- the trial involved 24 children with symptomatic SMN1-related proximal spinal muscular atrophy (SMA), aged between 1.5 and 9 years, weighing between 8.5 and 21 kg, 87.5% of whom had stopped other innovative therapies prior to inclusion.
- after one year, motor function was maintained or improved in most participants, with an increase in RULM and HFMSE scores.
- a large majority (95.8%) of participants who could sit with light support maintained this ability until the end of the study.
- three patients acquired the ability to stand with support and one to walk with support.
- the six participants who could walk at the start of the trial were still walking at the end of the study.
- transient asymptomatic transaminase elevations or thrombocytopenia were observed in more than half the participants and were treated appropriately.
Novartis presents new data on safety and efficacy of Zolgensma, including maintained and improved motor milestones in older and heavier children with SMA. Novartis press release, 4 March 2024.
