Roche Highlights 2-Years Data from RAINBOWFISH Trial of Evrysdi for Treating Children with Spinal Muscular Atrophy (SMA) at WMS 2024
Shots:
- Roche reported 2yrs. analysis of RAINBOWFISH trial (carried out with SMA Foundation & PTC Therapeutics), assessing Evrysdi (risdiplam) in SMA children (n=23) treated pre-symptomatically as infants
- All children with ≥3 SMN2 copies (n=18) were able to stand & walk; among those with 2 SMN2 copies (n=5), all were able to sit & 60% could stand & walk independently; all of them could swallow & feed orally without requiring permanent ventilation post 2yrs. They also demonstrated cognitive skills similar to non-SMA
- No deaths or AEs led to treatment discontinuation; common AEs included teething, gastroenteritis, diarrhea, eczema & fever, consistent with other Evrysdi trials & were related to age than SMA, that resolved over time
Ref: Roche | Image: Roche
Related News:- Roche's Evrysdi Receives the EC’s Marketing Authorization Extension for Newborns with Spinal Muscular Atrophy
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
