Spinal muscular atrophy type 2 gene therapy long-term data with pediatric neuromuscular disease treatment
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Novartis Reports Long-Term Data for Zolgensma in Spinal Muscular Atrophy Type 2

Novartis announces long-term follow-up data for onasemnogene abeparvovec (Zolgensma) in patients with spinal muscular atrophy type 2.

Novartis today announced long-term follow-up data for Zolgensma (onasemnogene abeparvovec) in patients with spinal muscular atrophy (SMA) type 2. At 3 years, patients treated with Zolgensma maintained motor function improvements achieved after treatment, with no new safety signals identified. These long-term data demonstrate the durable benefit of Zolgensma gene therapy in SMA type 2 and provide important evidence for the long-term safety and efficacy of this one-time treatment in older SMA patients.

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