Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse.

BACKGROUND:Duchenne muscular dystrophy (DMD) is a lethal, progressive muscle wasting disease caused by a loss of sarcolemmal bound dystrophin, which results in the death of the muscle fibers leading to the gradual depletion of skeletal muscle. There is significant evidence demonstrating that increas...

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Main Authors: Jonathon M Tinsley, Rebecca J Fairclough, Richard Storer, Fraser J Wilkes, Allyson C Potter, Sarah E Squire, Dave S Powell, Anna Cozzoli, Roberta F Capogrosso, Adam Lambert, Francis X Wilson, Stephen P Wren, Annamaria De Luca, Kay E Davies
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2011-05-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3089598?pdf=render