Clinical Research Directory
Browse clinical research sites, groups, and studies.
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
Sponsor: Hoffmann-La Roche
Summary
This multicenter, non-interventional, prospective study with a retrospective component aims to evaluate the real-world clinical outcomes, safety profile, and motor function improvements of Risdiplam treatment in participants with Type I and Type II Spinal Muscle Atrophy over 24 months of treatment. The study will allow retrospective collection of medical history data and previous participants visits from the medical records.
Official title: A Non-lnterventional, Multi-Center Study Investigating the Outcomes of Risdiplam in Type I and Type II Spinal Muscle Atrophy Patients
Key Details
Gender
All
Age Range
Any - 5 Years
Study Type
OBSERVATIONAL
Enrollment
30
Start Date
2026-04-15
Completion Date
2029-03-01
Last Updated
2026-04-15
Healthy Volunteers
No