Most Evrysdi-treated children walking after two years
Roche (ROG: SIX) has presented positive two-year data from the ongoing RAINBOWFISH study assessing the efficacy and safety of Evrysdi (risdiplam) in children with spinal muscular atrophy (SMA) who were treated pre-symptomatically as infants before six weeks of age.
The study found the majority of children achieved key motor milestones, were able to swallow and feed orally, and demonstrated cognitive skills typical of children without SMA, with none requiring permanent ventilation.
These data were presented at the 29th World Muscle Society (WMS) Congress in Prague, Czech Republic.
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