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About Duchenne

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About Duchenne

Duchenne muscular dystrophy, often referred to as DMD, or simply Duchenne, is the most common form (of more than 30 types) of muscular dystrophy.

Muscular dystrophy is an overarching term for a group of muscle diseases, defined by muscle weakness. The Canadian Neuromuscular Disease Registry (CNDR) estimates there are more than 800 boys and young men in Canada currently living with Duchenne muscular dystrophy and coping with its debilitating symptoms.

Receiving a diagnosis of Duchenne can be overwhelming, but we are here to make the journey a little easier for families who are searching for a more hopeful way forward. Our organization wants to connect you with the right tools to help you navigate your journey and ensure you know you are not alone.

We believe that when it comes to dealing with Duchenne, knowledge is not only power, but it is a source of comfort for families who are searching for answers that may change the trajectories of their sons’ lives.

Explore our site to learn more about what Duchenne is, how it affects those who have it and what your options for treatment are. If you have any questions about our web content or want to connect with other families who are on similar journeys, please do not hesitate to reach out.

Duchenne Basics

Duchenne muscular dystrophy is an extremely complex disease with a broad range of progressive symptoms. This section is intended to explain the what, why and how behind the most common form of muscular dystrophy diagnosed in children.

Diagnosis

Early diagnosis of Duchenne muscular dystrophy is the best way to enhance your child’s short and long-term outcomes. Learn more about the most common signs and symptoms and the types of testing available to support your journey to diagnosis.

Read the latest in Research and Drug Development

PepGen has announced the results from their completed Phase 1 healthy normal volunteer (HNV) trial of PGN-EDO51, the company’s lead exon skipping product for Duchenne muscular dystrophy.
Roche is now recruiting caregivers and patients who are able to offer their perspectives on completing the North Star Ambulatory Assessment (NSAA) or Performance of Upper Limb (PUL) as part of a new research study.
Edgewise Therapeutics has announced positive 4-month interim results from the ARCH open label study of EDG-5506 in adults with Becker muscular dystrophy (BMD).