About Action duchenne
Action Duchenne: A Leading Charity for Duchenne Muscular Dystrophy
Action Duchenne is a national charity that was established in 2001 with the aim of supporting individuals and families affected by Duchenne muscular dystrophy (DMD). As the first charity dedicated to this cause, Action Duchenne has been at the forefront of research, education, and advocacy efforts to improve the lives of those living with DMD.
Duchenne muscular dystrophy is a genetic disorder that primarily affects boys. It causes progressive muscle weakness and wasting, leading to difficulty walking, breathing, and performing everyday tasks. There is currently no cure for DMD, but there are treatments available that can help manage symptoms and slow down disease progression.
Action Duchenne's mission is to accelerate research into effective treatments for DMD while providing support and resources for those affected by the condition. Over the years, Action Duchenne has invested over £10m in groundbreaking research projects aimed at developing new therapies for DMD.
One of Action Duchenne's most significant achievements has been its role in funding clinical trials for exon skipping drugs. These drugs work by "skipping" over faulty sections of DNA in patients with specific mutations that cause DMD. This approach has shown promising results in improving muscle function and slowing down disease progression.
In addition to funding research projects, Action Duchenne also provides educational programs and resources for individuals living with DMD and their families. The charity offers workshops on topics such as managing symptoms, accessing healthcare services, navigating school systems, and planning for future care needs.
Action Duchene also advocates on behalf of individuals living with DMD at local, national, and international levels. The charity works closely with policymakers to ensure that people affected by rare diseases like DMD have access to appropriate care services.
Overall Action duchene plays an essential role in supporting those living with duchene muscular dystrophy through its investment into ground-breaking research, educational programs, and campaigns. The charity's work has helped to improve the lives of countless individuals and families affected by DMD, and its ongoing efforts continue to offer hope for a brighter future.
actionduchenne.org
