Roche: positive data in spinal muscular atrophy

(CercleFinance.com) - Roche reports positive two-year data from the ongoing Rainbowfish study, evaluating its Evrysdi (risdiplam) in children with spinal muscular atrophy (SMA) who were presymptomatically treated as infants before six weeks of age.


According to the study, 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.

The Swiss healthcare group adds that there were no deaths, nor any adverse events that led to treatment discontinuation, with the most common adverse events being teething, gastroenteritis, diarrhea, eczema and pyrexia.

These data were presented at the 29th Congress of the World Muscle Society (WMS), held from October 8 to 12. Roche is leading the clinical development of Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics.

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