Duchenne muscular dystrophy

J Paediatr Child Health. 2015 Aug;51(8):759-64. doi: 10.1111/jpc.12868. Epub 2015 Mar 9.


Duchenne muscular dystrophy, an X-linked disorder, has an incidence of one in 5000 boys and presents in early childhood with proximal muscle weakness. Untreated boys become wheelchair bound by the age of 12 years and die of cardiorespiratory complications in their late teens to early 20s. The use of corticosteroids, non-invasive respiratory support, and active surveillance and management of associated complications have improved ambulation, function, quality of life and life expectancy. The clinical features, investigations and management of Duchenne muscular dystrophy are reviewed, as well as the latest in some of the novel therapies.

Keywords: Duchenne; corticosteroid; gene therapy; muscular dystrophy; non-invasive ventilation; positive pressure.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adolescent
  • Child
  • Humans
  • Male
  • Molecular Sequence Data
  • Muscular Dystrophy, Duchenne* / drug therapy
  • Muscular Dystrophy, Duchenne* / genetics
  • Muscular Dystrophy, Duchenne* / physiopathology

Associated data

  • GENBANK/CD003725