There are currently four types of research underway.

  • 1. Exon Skipping Research - a type of gene therapy studying drugs that enable the body to skip over genetic defects to produce dystrophin, causing DMD to become a less serious, Becker form of MD.

  • 2. Research on Currently Approved FDA Drugs - using drugs like sildenafil to perhaps increase heart functionality.

  • 3. New Drug Research - exploring currently unknown medicinal ways to target the different and varying aspects of the disease.

  • 4. Stem Cell Research - exploring how to take blood vessel cells from matched siblings to override the genetic effect of DMD.

Read more about current research and trials using the below links.

List of Trials from Treat-NMD


Cure Duchenne

Duchenne Ireland

Nationwide Children's


U.S. Nat'l Library of Medicine


 Aidan as Race Director
DMD Explained
DMD is a genetically caused disease, mostly in boys, whereby the body is missing a large protein called dystrophin. This protein enables the body to regenerate all body muscles as they're subject to wear and tear. Boys with DMD are unable to produce dystrophin, causing their muscles to deteriorate over time.