Duchenne Muscular Dystrophy (DMD) is a devastating muscle-wasting disease. It is the most common and severe form of Muscular Dystrophy. Diagnosed in childhood, it mainly affects boys. There is currently no cure. Started by families affected by the disease, Duchenne UK has one clear aim – to end Duchenne.

We’re doing this by funding research that’s focused on getting treatments to those affected now – as well as pushing for an effective treatment in the future.

We connect leading researchers with industry, the NHS and patients to challenge every stage of drug development, from research to clinical trials to drug approval. We connect families with each other to create a network of mutual support and to pool our resources, knowledge and experience. We connect brilliant people and generous philanthropists to grow our impact and help them be part of something incredible – the end of Duchenne.

Together we’ve created new infrastructure to deliver clinical trials through the DMD Hub, and we fund doctors, nurses, physiotherapists and trial coordinators needed to run trials. We’ve uncovered treatments and enabled them to be delivered to patients. We’ve successfully campaigned to enable quicker access to new treatments. And through Project HERCULES, we have established an innovative way to connect leading DMD drug developers to collaborate to speed up access to treatments.

Our heart and soul, and our reason for carrying on, are our boys and men, and rare girls, living with DMD.

This collaborative approach means that we are able to focus on ending Duchenne as quickly and effectively as possible.

And it means you can be part of it too.

Together, we will end Duchenne.

We have achieved some incredible results, with the help of donors, partner charities and researchers, for details please read:  Our Impact.

We have an innovative approach to funding. Not only do we fund basic research, but we fund clinical trials. We fund the doctors and nurses in the UK to deliver those trials: and we look at where ever possible, we can accelerate research and work with industry and regulators to get drugs approved.

To achieve our mission, we adopt an integrated approach that has 3 main areas of activity/focus.

1) Funding Research and Trials:

Our approach is sharply focused on advancing research – and getting treatments into the clinic and to patients as quickly as possible. We actively seek out projects and possible therapies that could benefit this generation of patients. We invest globally in both basic science and translational research.

Read more here about the research projects and clinical trials that we fund. 

2) Accelerating Drug Development:

Funding the research is the first crucial step. But funding research on its own it isn’t enough. So we proactively invest in every stage of the drug development timeline to improve and accelerate the process.

Read more here about how we accelerate drug development through our own initiatives and by working both globally and in the UK with industry, governments and health providers. 

3) Community Engagement & Support:

Patients with DMD are at the heart of everything we do. Duchenne UK was set up by parents to fight every day to speed up treatments for all those living with DMD. Our drive and ambition comes from our deep understanding of the pain and the challenges that families face. 

Read here about how we engage with the Duchenne community and collaborate with other charities to improve the life of those living with Duchenne.

Duchenne UK is a member of the AMRC.

READ OUR IMPACT REPORT 2019