Roche announces results from Evrysdi (risdiplam) study in infants with Type 1 spinal muscular atrophy (SMA) published in New England Journal of Medicine
FIREFISH Part 1 data show treatment with Evrysdi at 12 months helped 90% of these infants survive without permanent ventilation and 33% sit without support, a key motor milestone not normally seen in the natural course of the disease The FDA approved Evrysdi in August 2020 as the first and only at home SMA treatment... Read more