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).
The charity has a strong record in funding research and has to date funded 8 major projects costing £800,000 and has lead the £1.6m DoH funding of the MDEX project. These projects have enabled much needed early work to be completed on exon skipping and other therapeutic approaches.
Action Duchenne holds an international conference every year to bring together researchers and families to exchange new research developments and provide a vital meeting venue for scientists.
In 2005 Action Duchenne launched the Duchenne Registry, the first National Duchenne database that holds gene information of people living with Duchenne and can be used to speed up the recruitment of patients for clinical trials.
In 2006 Action Duchenne launched a comprehensive learning and behaviour toolkit for use by parents and education professionals.
Celebrity supporters 2
Action Duchenne has received support from the following celebrities listed on this site: