Spinal muscular atrophy gene therapy long-term outcomes with AAV9 vector delivery and rare disease research
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Novartis Presents 5-Year Long-Term Data for Zolgensma in Spinal Muscular Atrophy

Novartis announces 5-year long-term data showing Zolgensma (onasemnogene abeparvovec) maintains motor function improvements in children with spinal muscular atrophy.

Novartis today presented 5-year long-term data from the START and STR1VE trials showing that Zolgensma (onasemnogene abeparvovec) maintains significant motor function improvements in children with spinal muscular atrophy (SMA) type 1. At 5 years, all patients who received Zolgensma in the START trial were alive and event-free, compared to a natural history where most patients with SMA type 1 die or require permanent ventilation by 2 years of age. These long-term data demonstrate the durable benefit of Zolgensma and support its use as a one-time gene therapy for SMA.

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