Overview

A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Status:
Recruiting
Trial end date:
2029-01-21
Target enrollment:
Participant gender:
Summary
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
Phase:
Phase 2
Details
Lead Sponsor:
Hoffmann-La Roche
Treatments:
Risdiplam