SRP-4053

DRUG3 trials

Sponsors

Sarepta Therapeutics, Inc.

Conditions

Duchenne Muscular DystrophyDuchenne muscular dystrophy

Phase 1

Phase I/II Study of SRP-4053 in DMD Patients

CompletedNCT02310906

Sarepta Therapeutics, Inc.Duchenne Muscular Dystrophy

Start: 2015-01-13End: 2019-03-25Updated: 2020-10-19

Phase 3

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

CompletedNCT02500381

Sarepta Therapeutics, Inc.Duchenne Muscular Dystrophy

Start: 2016-09-28End: 2025-10-16Updated: 2025-11-18

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc.Duchenne muscular dystrophy

Target: 4Updated: 2024-02-28

Related Papers

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Nucleic Acid Therapeutics2021-11-17114 citations

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping

JAMA Neurology2020-05-26250 citations

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.

2020-03-05246 citations