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 increa...

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Bibliografski detalji
Glavni autori: Tinsley, J, Fairclough, R, Storer, R, Wilkes, F, Potter, A, Squire, SE, Powell, D, Cozzoli, A, Capogrosso, R, Lambert, A, Wilson, F, Wren, S, De Luca, A, Davies, K
Format: Journal article
Jezik:English
Izdano: Public Library of Science 2011