Neuroscience

AAN Presentation 2019: Update from SUNFISH part 1: Safety, tolerability and PK/PD from the dose-finding study, including exploratory efficacy data in patients with Type 2 or 3 spinal muscular atrophy (SMA) treated with risdiplam (RG7916)

May 07, 2019 by [E Mercuri, G Baranello, J Kirschner, L Servais, N Goemans, MC Pera, M Tichy, WY Yeung, H Kletzl, M Gerber, C Czech, Y Cleary, K Gorni and O Khwaja]

SUNFISH (NCT02908685) is a multicenter, two-part, randomized, placebo-controlled, double-blind Phase 2 study evaluating the efficacy and safety of risdiplam (RG7916) in patients with Type 2 or Type 3 SMA aged 2-25 years. This presentation describes safety, tolerability and PK/PD data from all patients in SUNFISH Part 1 and exploratory efficacy data in patients treated with risidplam for ≥1 year.

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