Action Duchenne is a charity with a simple vision - a world where no one is limited by Duchenne muscular dystrophy.
Duchenne muscular dystrophy is a rare genetic condition which causes mutations in the dystrophin gene. This prevents the production of dystrophin, which is a vital muscle protein. The lack of dystrophin means that muscles are more susceptible to damage and waste away over time. Many individuals with the condition require a powered wheelchair from their early teens, and the condition eventually affects the heart and breathing muscles. Sadly, there is no cure for Duchenne. The life expectancy of the condition is around 30 years, but this has improved thanks to developments in palliative care.
What Does Action Duchenne Do?
Action Duchenne has three core objectives to help them towards their vision:
Develop effective treatments for Duchenne - The charity fund research, educate clinicians and support clinical trials in order to develop more effective treatments for the condition.
Build a community - By uniting families and educating people on the condition, more people can be made aware of Duchenne.
Strive for a more inclusive society - The charity promotes equality and the day to day acceptance and accessibility for those with Duchenne.
50% of Action Duchenne’s staff and trustees have children living with the condition, meaning that they can provide on-point expertise and support for families and individuals affected.
Over the years, Action Duchenne have had major successes in the world of medical research that have helped bring hope to families living with the condition. Scientists have developed ‘exon skipping’ drugs, while the charity’s disease registry has put the UK at the forefront of clinical clinical trials. Action Duchenne have also provided research funding that has given over 100 people the chance to take part in life-changing clinical trials. Currently, the charity is funding a gene therapy clinical trial which will enable more families to be given the opportunity to take part.
Action Duchenne have support officers who provide vital support to over 1000 families every year, and they aim to help every single person and family affected by the condition. This can be helping families to apply for benefits, attending school meetings or simply providing a listening ear when needed.
Duchenne parents often say that they only feel understood by another Duchenne parent, so the charity works to bring families together so they can provide vital support to one another. Additionally, they organise the largest Duchenne conference in Europe, which helps to unite families with researchers, clinicians and drug developers working in the field.
How Can You Support Action Duchenne?
This January, we are supporting Action Duchenne in our charity poll, and you can help them to win 20% of our profits in our first poll of 2021. Every penny raised helps the charity to fund cutting edge research and clinical trials that will go towards making a difference to families and individuals affected by Duchenne. Cast your vote for them today and help fund life-changing research to help bring hope to more families.
Find out more about Action Duchenne and how you can support their work further by visiting their website: https://www.actionduchenne.org.