October 4, 2023- Roche presented positive results from the primary analysis of the ongoing RAINBOWFISH study assessing the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic SMA, aged from birth to six weeks.
The data were presented at the 28th World Muscle Society (WMS) Congress, October 3-7, 2023. Clinical studies show that the loss of motor neurons may begin before symptoms start so initiating treatment early is critical for better outcomes. The RAINBOWFISH study included babies with two or more copies of the SMN2 gene. Generally, the lower the number, the more severe the disease.
RAINBOWFISH was the first trial to assess cognition with a standardised scale (BSID) as an exploratory endpoint and results showed cognitive skills typical of normal child development after 1 year of Evrysdi treatment, assessed by BSID-III.