Duchenne Muscular Dystrophy
by Amy Pohler and Kaitlyn Eschler
Facts
- Duchenne muscular dystrophy is an inherited disorder that involves muscle weakness, which quickly gets worse.
- Most common fatal genetic disorder diagnosed in childhood.
- Because Duchenne gene is found on X-chromosome it primarily affects boys.
- Those affected only live into late 20's.
Treament
- There is no known cure to Duchenne muscular Dystrophy.
- Steroid drugs can slow the loss of muscle strength.
Prognosis
- Duchenne muscular dystrophy leads to progressive worsening disability.
- Death typically occurs from lung disorders.
Little Known Fact
There is an experimental drug — known as drisapersen — used in a clinical trial and is designed to effectively cover over the specific genetic mutation, allowing the problem area to be skipped and causing cells to produce a slightly shorter – but functional – dystrophin protein.