A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
Status:
Active, not recruiting
Trial end date:
2024-12-27
Target enrollment:
Participant gender:
Summary
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the
safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants
with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the
splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.