Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)
- Exploratory efficacy data showed 88% of infants treated with Evrysdi were alive and did not require permanent ventilation at two years
- 59% of infants were able to sit without support for at least 5 seconds
- No new safety signals were identified
- In August, the FDA approved Evrysdi for the treatment of SMA in adults and children 2 months and older
For the full text by Roche please click here.