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Neurology

Investigators at Cleveland Clinic target neurology

Published in Gene Therapy Weekly, September 2nd, 2010

Scientists discuss in 'Targeting fibrosis in Duchenne muscular dystrophy' new findings in neurology. "Duchenne muscular dystrophy (DMD) is the most common genetic muscle disease affecting 1 in 3,500 live male births. It is an X-linked recessive disease caused by a defective dystrophin gene," scientists writing in the Journal of Neuropathology and Experimental Neurology report.

"The disease is characterized by progressive limb weakness, respiratory and cardiac failure, and premature death. Fibrosis is a prominent pathological feature of muscle biopsies from patients with DMD. It directly causes muscle dysfunction and contributes to the lethal DMD phenotype....

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