Connect to Clinical Trials

With over 35 companies working on new therapies for Duchenne, you may be approached about participating in a clinical trial. Often, these conversations arise with one of your child’s neuromuscular care team clinicians, a pharmaceutical company representative, or through another family with a child with Duchenne. Navigating clinical trial participation is a very complex system, requiring extensive education and understanding of the process. PPMD aims to provide you with the tools to make the best informed decision for your family.

Find out About Research Studies & Clinical Trials

The Duchenne Registry is PPMD’s patient-report registry for Duchenne and Becker muscular dystrophy. This international registry began in 2007 and now has over 4,000 members. The Duchenne Registry connects Duchenne and Becker patients with actively recruiting clinical trials and research studies, and educates patients and families about Duchenne and Becker research. At the same time, The Duchenne Registry is a valuable resource for researchers, allowing access to data provided by patients and their families—information that is vital to advances in the care and treatment of Duchenne. To learn more and to register, please visit DuchenneRegistry.org.

Know your child’s genetic mutation

It is an incredibly exciting time in the Duchenne community with so many potential therapies in the pipeline. Many of these therapies are mutation-specific, meaning they will only work for individuals with certain mutations in their Duchenne gene. If you’ve never had genetic testing or if you need repeat genetic testing, our Decode Duchenne program provides FREE genetic testing to eligible individuals.

Explore PPMD’s interactive drug development pipeline

PPMD provides a variety of resources for learning about potential treatments and clinical trials for people with Duchenne and Becker, as well as female carriers. PPMD developed an interactive drug development pipeline to further help the community stay up-to-date on all therapies that are currently in development. Click here to explore investigational drugs by therapeutic approach.