Neuroscience

JEWELFISH (NCT03032172) is a multicentre, open-label study primarily evaluating the safety and tolerability of once-daily oral administration of risdiplam (RG7916) in patients aged 12–60 years with Type 2 or 3 SMA who have previously participated in a study with therapy targeting SMN2 splicing. Primary endpoints for Part 1 are safety, tolerability and PK/PD of risdiplam.