Pilot Study Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
Latest Information Update: 15 Apr 2025
At a glance
- Drugs Nusinersen (Primary) ; Onasemnogene-abeparvovec (Primary) ; Risdiplam (Primary)
- Indications Spinal muscular atrophy
- Focus Therapeutic Use
Most Recent Events
- 10 Apr 2025 Planned initiation date (estimated date of first participant enrollement) changed from 1 Apr 2025 to 1 May 2025.
- 05 Mar 2025 Planned initiation date (estimated date for recruitment of the first subject) changed from 1 Feb 2025 to 1 Apr 2025.
- 05 Mar 2025 Status changed from not yet recruiting to recruiting.