Mission

Our mission is to direct money into the hands of the researchers who have the very best chance of finding a cure or treatment for Duchenne Muscular Dystrophy. Our goal is to cure DMD before it takes Harrison's life and the lives of thousands of boys like him. Whilst there are many wonderful charities out there who work so hard at raising funds for Duchenne, the money donated is usually put towards subsidising the tremendous cost of care rather than cure. These organisations provide an incredible service for the young boys suffering from this cruel disease as well as offering assistance to their families. However, if we are to conquer DMD, we need to also look into researching a cure or effective treatment. Alongside a few organisations, we invest our money in translational research that focuses on moving science from the lab into human clinical trials so that finding a cure becomes more real allowing the little boys with DMD to grow into the strong men they are meant to be.

Category

Health

Additional Information

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. The disease almost always affect boys, and they tend to be diagnosed before the age of 5. Duchenne muscular dystrophy is classified as a rare disease. There are around 2,500 patients in the UK and an estimated 300,000 sufferers worldwide. Children born with Duchenne muscular dystrophy have a fault, known as a mutation, on their dystrophin gene, the longest gene in the body. The fault means that they cannot produce dystrophin, a protein that is vital for muscle strength and function. This lack of dystrophin results in a progressive deterioration of muscle strength and function.