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Roche: efficacy of Evrysdi in spinal muscular atrophy

( - Roche has announced five-year data confirming the efficacy and safety of Evrysdi (risdiplam) in children with type 1 spinal muscular atrophy.

At the end of the fifth year, 91% of treated children were alive, 81% without permanent ventilation, and 59% could sit without support.

Seven children could stand and some could walk with support. Evrysdi helped maintain or improve motor skills, swallowing and tube-free feeding, the laboratory points out.

Adverse events fell by 66% between the first and last years, with fewer hospitalizations.

Evrysdi is the only non-invasive oral treatment for SMA, developed by Roche in collaboration with the SMA Foundation and PTC Therapeutics.

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