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NOT YET RECRUITING
NCT07531719

A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)

Sponsor: Hoffmann-La Roche

View on ClinicalTrials.gov

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