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 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.
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.
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Please read about the progress towards our shared vision: of a future without Duchenne muscular dystrophy, made throughout the past three years, all made possible by our loving, dedicated supporters.
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PepGen has received clearance by Health Canada for the CONNECT1-EDO51 Phase 2 clinical trial of PNG-EDO51, an exon 51 skipping therapy.