Duchenne muscular dystrophy (DMD, MIM # 310200) is an X-linked recessive condition affecting approximately one in 5000 live male births [1]. It is caused by mutation in the DMD gene (MIM # 300377) that leads to the absence or disruption of expression of the protein called dystrophin, found in a variety of tissues including skeletal and cardiac muscle and the central nervous system (CNS). A number of transcripts are produced from the DMD gene; these encode various dystrophin isoforms, some of which are expressed in the brain.

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