Dedicated to funding and accelerating treatments for
Duchenne Muscular Distrophy

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. The disease almost always affect boys, and they tend to be diagnosed before the age of 5. Duchenne muscular dystrophy is classified as a rare disease. There are around 2,500 patients in the UK and an estimated 300,000 sufferers worldwide.

Children born with Duchenne muscular dystrophy have a fault, known as a mutation, on their dystrophin gene, the longest gene in the body. The fault means that they cannot produce dystrophin, a protein that is vital for muscle strength and function. This lack of dystrophin results in a progressive deterioration of muscle strength and function.

Duchenne UK's mission

Duchenne UK is run by parents, fighting every day to speed up treatments for all our children and those living with DMD. Our sons have taken part in clinical trials. We understand the pain and the challenges families face. And we are fighting for all of us. We can learn from our experiences on the front line, because we live with Duchenne every minute of every day. And we can act. Read about what we did here to address clinical trial capacity in the UK.

Patients with DMD are at the heart of everything we do.

Our approach is 100% 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.
Our Scientific Advisory Board comprises scientists, doctors and drug developers so that we look at each project and ask: Is this good science? Can this be taken into the clinic? What hurdles exist? Is this being replicated anywhere else?

Find out more about Duchenne Charity UK here