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!

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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

March 13, 2024
Following the FDA approval on October 26, 2023, AGAMREE is now available by prescription and dispensed throughout the United States through a specialty pharmacy network.
March 13, 2024
PepGen shares a press release announcing the FDA has granted both orphan drug and rare pediatric disease designations for PGN-EDO51.
March 5, 2024
REGENXBIO reports additional interim safety and efficacy data in the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in patients with Duchenne.
February 16, 2024
Sarepta announces the U.S. Food and Drug Administration (FDA) has accepted and filed the Company's efficacy supplement to the Biologics License Application for ELEVIDYS.
February 13, 2024
Edgewise shares a press release announcing FDA granting Fast Track designation for EDG-5506 for the treatment of Duchenne.
February 8, 2024
REGENXBIO announces completion of enrollment in the Phase I/II AFFINITY DUCHENNE® trial of RGX-202.

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