Conference report: Positive 1 year data from Part 2 of SUNFISH study evaluating risdiplam in people aged 2-25 years with nonambulatory type 2 and type 3 spinal muscular atrophy (SMA)

Study (n=180) showed that change from baseline in primary endpoint Motor Function Measure scale (MFM-32) was significantly greater in patients receiving risdiplam compared to placebo (1.55 point mean difference; p=0.0156) at 12 months of treatment.


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