Harrisons Fund

the disease

Duchenne Muscular Dystrophy is a stinker of a disease. It's the most common fatal genetic disorder to affect children around the world. If you've got it, you can't produce dystrophin, a protein you need to build up your muscles. As a result, every muscle in the body deteriorates. At the moment there is no cure.

The facts about Duchenne are inescapable:

  • Duchenne Muscular Dystrophy is 100% fatal
  • Most kids with it die in their late teens or early twenties
  • Most with it are usually in a wheelchair by the age of 12
  • It leads to respiratory failure, heart failure, and other debilitating orthopaedic complications

One in 3,500 boys is born with it, and in the UK 2,500 kids has it at the moment. You can have it, no matter where you are or what your ethnic background is. A third of all cases start in the womb, with no warning before the baby is born. Girls can also get Duchenne with around 1% of Duchenne births being female.

Although we know a lot about how Duchenne is caused, current treatments are pretty limited. Steroids and daily stretching are what most doctors recommend, but they have their drawbacks and don't prevent debilitating muscle contractures.

tweety-pie

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