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: 14 Jul 2026
At a glance
- Drugs Nusinersen (Primary) ; Onasemnogene abeparvovec (Primary) ; Risdiplam (Primary)
- Indications Spinal muscular atrophy
- Focus Therapeutic Use
Most Recent Events
- 09 Jul 2026 Planned End Date changed from 1 Mar 2027 to 1 Sep 2028.
- 09 Jul 2026 Planned primary completion date changed from 1 Sep 2026 to 1 Sep 2027.
- 10 Oct 2025 Planned initiation date changed from 1 Oct 2025 to 1 Nov 2025.