This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability,
efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with
deletions amenable to exon 53 skipping.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Sarepta Therapeutics Sarepta Therapeutics, Inc.
Collaborators:
Catholic University of the Sacred Heart Consultants for Research in Imaging and Spectroscopy Great Ormond Street Hospital for Children NHS Foundation Trust Institut de Myologie, France Royal Holloway University Sysnav University College, London University of Newcastle Upon-Tyne