Tag: Evrysdi (risdiplam)
New clinical trial to Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)
Roche presents its new data on the SUNFISH, JEWELFISH, RAINBOWFISH study
- 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
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