Clinical Trials

For a quick look at enrolling or soon to be enrolling investigational drug studies in Duchenne and their enrollment criteria, check out our Clinical Trial Cheat Sheet below.

Clinical Trial Finder

Use this Clinical Trial Finder below that can help provide you with more information regarding clinical trials in Duchenne that that you, your child, or your patient may be eligible for!

Watch Jett Foundation's Mini Satellite Presentation

Presented by Jean Mah, M.D., Associate Professor of Pediatric Neurology Alberta Children’s Hospital at the 2016 Child Neurology Society Meeting to learn more about these potential therapies!

Latest Duchenne News

December 17, 2025
On December 16, 2025, The U.S. Department of Health and Human Services (HHS) Secretary Robert F. Kennedy, Jr. approved the addition of Duchenne Muscular Dystrophy (DMD) and Metachromatic Leukodystrophy (MLD)...
December 15, 2025
On December 1, 2025, Upsher-Smith Laboratories, LLC (Upsher-Smith) announced the launch of KYMBEE™ (deflazacort) Tablets, indicated for the treatment of Duchenne muscular dystrophy in patients 5 years of age and...
December 8, 2025
On December 8, 2025, Dyne Therapeutics, Inc. announced positive topline results from the Registrational Expansion Cohort (REC) of its Phase 1/2 DELIVER trial evaluating zeleciment rostudirsen (z-rostudirsen, also known as...
November 25, 2025
On November 25, 2025, Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort...
November 24, 2025
On November 24, 2025, Wave Life Sciences announced they have initiated Part C of FORWARD-53, a clinical trial evaluating the investigational molecule WVE-N531 in boys with Duchenne muscular dystrophy (DMD)...
November 21, 2025
On November 20, 2025, Avidity Biosciences shared a Community Letter detailing their definitive merger acquisition by Novartis. This letter comes after the official announcement on October 26, 2025.  To view...

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