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SUNFISH Parts 1 and 2: 5-year efficacy and safety data of risdiplam▼ in Types 2 and 3 spinal muscular atrophy (SMA)
SUNFISH was a multicenter, two-part, randomized, placebo-controlled, double-blind clinical trial of risdiplam▼ in a broad and heterogeneous population with Types 2 and 3 SMA (aged 2–25 years at enrollment). This is the first presentation of motor function and safety data at the end of the 5-year SUNFISH study. Children, teenagers and adults with SMA showed improvement or stabilization in motor function outcomes, which is in contrast to the decline observed in an untreated population.
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