Pilot Study Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies

Status: Recruiting

Phase of Trial: Phase IV

Latest Information Update: 10 Mar 2025

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Drugs Nusinersen (Primary) ; Onasemnogene-abeparvovec (Primary) ; Risdiplam (Primary)
Indications Spinal muscular atrophy
Focus Therapeutic Use

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.
13 Jan 2025 Planned initiation date changed from 1 Jan 2025 to 1 Feb 2025.

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