Overview

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Status:
Not yet recruiting

Trial end date:
2028-03-31

Target enrollment:

Participant gender:

Summary

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children < 2 years of age genetically diagnosed with SMA.

Phase:

Phase 4

Details

Lead Sponsor:

Hoffmann-La Roche

Treatments:

Risdiplam