The charity was the first organisation in the UK dedicated exclusively to Duchenne and Becker Muscular Dystrophy and, with the help and support of friends and supporters and other Duchenne families, developed into a national organisation.
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births (around 2500 people have DMD in the UK).
Action Duchenne’s main aim has always been to find a cure for Duchenne and Becker through fundraising and campaigning to raise awareness and develop the necessary protocols. However, there needs to be a dual strategy: searching for a cure whilst, at the same time, ensuring that everyone was gets the standard of care necessary to have the best life possible. This not only includes best medical care from qualified professionals in Centres of Excellence, but educational and social care so that young people with Duchenne and their families can look forward to a brighter future that involves good job or training prospects, independent living options and necessary support.
Four Areas of Focus:
Cure: To change Duchenne into a treatable and manageable condition by producing, and delivering, a broad research strategy that seeks to find the cure or treatment for Duchenne or Becker muscular dystrophy.
Influence: To inform political debate in the fight to improve the quality of life for those living with Duchenne or Becker muscular dystrophy by changing attitudes in standards of care, healthcare professionals and to influence the bench to bed times for treatments for those suffering with rare conditions.
Empower: To continue to provide education for those living with duchenne/beckers muscular dystrophy through Decipha and Takin’ Charge by: continuing to fund Decipha to provide specialist learning assessments and interventions to support young people , their families and schools and to increase attendance at the International Conference.
Improve: To make the DMD registry as the database of choice for pharma and clinicians by increasing the numbers of those registering on the database and to increase revenue from it.