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A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy — Jewelfish  

Spinal Muscular Atrophy Clinical Trial

Official title:
An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric Patients With Spinal Muscular Atrophy

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03032172
Study type Interventional
Source Hoffmann-La Roche
Contact
Status Active, not recruiting
Phase Phase 2
Start date March 3, 2017
Completion date December 27, 2024
View Details
See also
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