Early Treatment Shows Benefit in Infants With Presymptomatic Genetic Disease



(MedPage Today) — Infants up to 6 weeks old with genetically diagnosed presymptomatic spinal muscular atrophy (SMA) treated with oral risdiplam (Evrysdi) appeared to have better functional and survival outcomes compared with a natural history…



Source link : https://www.medpagetoday.com/neurology/generalneurology/116978

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Publish date : 2025-08-13 21:00:00

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