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SUNFISH PARTS 1 AND 2: 4-YEAR EFFICACY AND SAFETY DATA OF RISDIPLAM▼ IN TYPES 2 AND 3 SPINAL MUSCULAR ATROPHY (SMA)
SUNFISH (NCT02908685) is a multicenter, two-part, randomized (2:1, risdiplam▼:placebo), placebo-controlled, double-blind study evaluating the efficacy and safety of risdiplam▼ in patients with Type 2 or Type 3 SMA (inclusion criteria: 2–25 years at enrollment). This presentation will report safety and efficacy data from Parts 1 and 2 of the SUNFISH study after 4 years of treatment with risdiplam▼.
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