Duchenne muscular dystrophy is a rare, life-limiting, muscle-wasting condition, affects around 2,500 people in the UK, with the vast majority being male. Mutations, or changes, in the dystrophin gene stop the body making a vital muscle protein and over time this causes muscles to weaken and waste away.

Children, young people are usually diagnosed around four years old, and those living with the condition use a powered wheelchair in their teenage years, and most will require a ventilator in their twenties. 

Life expectancy is said to be 30 years. There is no cure. 

What have we achieved?

Formed in 2001, we were the first national charity dedicated to supporting those living with Duchenne. Our work to date has seen over £13m invested in ground-breaking research, educational programmes and campaigns and we have made some incredible achievements. These include:

• Our 'All-through Support' programme started in 2021, with the backing of the National Lottery Community Fund; the first of its type for Duchenne. Our programme offer free and accessible support to every UK family diagnosed with Duchenne. 
• We acted as a springboard for other patient groups including DMD Pathfinders – founded through our Takin’ Charge lottery funded project.
• We manage the UK DMD Registry. This database of people living with Duchenne has helped us increase the number of clinical trials in the UK.
• Our research enabled the development of a potential gene therapy for Duchenne. This will soon be tested in the Unite-DMD clinical trial.
• Our campaigning ensured Translarna, the first drug to treat an underlying cause of Duchenne, was made available across the UK.
• Our conference is the largest and best attended in the UK. It brings together hundreds of people from the Duchenne community including families, healthcare professionals, researchers and drug companies.
• Our social media channels have helped to educate a wider audience about Duchenne and ensure people are better informed about the condition.

We work along side other charities, the NHS, academia, biotech and drug companies. All to bring about the changes we want to see. We hugely value input from the Duchenne community, and telling us your stories can help us to help and educate others.