Action Duchenne

Who we are

Duchenne muscular dystrophy 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.

The first signs of Duchenne happen in the early years of life and it is usually diagnosed around 4 years old. The condition reduces life expectancy to an average of approximately 30 years and it has no cure.

Action Duchenne has a simple vision: a world where lives are no longer limited by Duchenne muscular dystrophy. 

We work to achieve our vision by: 

• funding research to identify and advance potential therapeutic strategies for everybody living with Duchenne, and support clinical trials and the staff and infrastructure required to bring trials to the UK

• campaigning for the NHS to provide their patients with the earliest possible access to medicines that have been approved for the treatment of Duchenne

• lobbying Government agencies to improve care and services offered to those affected by Duchenne

• striving to create an inclusive society where everybody is equal, where disability is accepted and is no longer a barrier in accessing transport and other facilities

• building a community for all those affected by Duchenne – to bring people together to share their experiences and to raise the profile of the condition. 

While we hope that research and clinical trials will one day lead to a treatment, or cure, for Duchenne, we believe it is vital to support families living with the condition today. We support families from diagnosis through their whole Duchenne journey.