Overview of the SUNFISH study
risdiplam
SUNFISH (NCT02908685) [Part 1 was a dose-finding study in patients with type 2 or type 3 SMA (ambulant and non‑ambulant) to inform the dose for Part 2. In Part 1, risdiplam treatment led to a sustained increase in SMN protein in the blood, and exploratory efficacy analyses showed improvement or stabilization in motor function [Here, we report longer-term exploratory efficacy and safety results after 24 months of risdiplam treatment in SUNFISH Part 2 and contextualize these findings with external comparator groups. In addition, we investigate the efficacy and safety of 1 year of risdiplam treatment in patients who previously received placebo up to month 12.