About the Charity
Action Duchenne (formally Parent Project UK) was set up by Duchenne families in 2001 to promote new research for a cure for Duchenne.
Duchenne affects 2,500 people in the UK and about 40,000 people worldwide. It has no cure and is caused by mutations in the dystrophin gene that prevent correct reading of the genetic code, the result of which is that the essential muscle protein dystrophin is not produced. This results in progressive muscle wasting, severely limiting mobility in teenage patients (boys and men) and leading to premature death in their twenties from respiratory or cardiac problems (the heart muscle is directly affected by the genetic defect).
How to Get Involved
New Gene Therapy approaches are being trialled in labs in the UK that might in the future be able to fix or replace the faulty gene and so bring about a treatment or cure for Duchenne. Drugs are also being tested that might help to delay the symptoms of the disease and give our sons longer and more active lives.
Action Duchenne aims to campaign to get funding partnerships involving the Government, trusts, other charities and money raised by Parents, families and supporters to take this promising lab work to clinical trials.
Your donation will help them to raise the £2 million they need over the next two years to accelerate research and raise awareness of Duchenne.
Helena Bonham Carter.
Get in Contact
If you want to get in contact with the charity, then visit the official site here!