Novartis' SMART study shows safety and efficacy of Zolgensma gene therapy for spinal muscular atrophy in older, heavier patients.

Novartis presents new data from the SMART study supporting the safety and efficacy of Zolgensma, the only one-time gene therapy for spinal muscular atrophy (SMA). The study, which included older and heavier SMA patients, demonstrated maintained or improved motor milestones for nearly all patients after 52 weeks. Zolgensma is now approved in over 51 countries, and more than 3,700 patients have been treated worldwide.

March 04, 2024
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