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.
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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.