Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
Status:
Completed
Trial end date:
2018-10-03
Target enrollment:
Participant gender:
Summary
This is a first-in-human dose-titration and open-label extension study to assess safety,
tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy
(DMD) patients with deletions amenable to exon 45 skipping.