WMS Poster 2018: SUNFISH Part 1: RG7916 treatment results in a sustained increase of SMN protein levels and the first clinical efficacy results in patients with Type 2 or 3 SMA
SUNFISH (NCT02908685) is a multicentre, randomized, placebo-controlled, seamless, Phase 2 study evaluating the efficacy and safety of risdiplam (RG7916) in patients with Type 2 or Type 3 SMA aged 2-25 years. Part 1 was dose finding for Part 2. Primary endpoints for Part 1 were safety, tolerability and PK/PD of risdiplam, whereas for Part 2 the primary endpoint is change from baseline in MFM32 at Month 12.
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