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...
Hoofdauteurs: | 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 |
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Formaat: | Journal article |
Taal: | English |
Gepubliceerd in: |
Public Library of Science
2011
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