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Novartis: drug shows benefit in SMA patients

(CercleFinance.com) - Novartis on Tuesday said that Zolgensma data has showed therapeutic benefit in patients with spinal muscular atrophy (SMA), including prolonged event-free survival.


In the study, nearly all (91%) patients met the co-primary efficacy endpoint of event-free survival after 14 months, the Swiss drugmaker said.

More than half (59%) of patients met the co-primary efficacy endpoint of sitting for more than 30 seconds at 18 months of age, a milestone never achieved in the natural history of this disease, which interferes with babies' ability to talk, eat, and even breathe.

In another trial assessing the efficacity of compound AVXS-101 in patients with Type 2 SMA, who are between two and five years of age, nearly all of them achieved a clinically meaningful response, consistently demonstrating improved motor function, Novartis added.

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