Second-generation compound for the modulation of utrophin in the therapy of DMD

Journal article

Duchenne muscular dystrophy (DMD) is a lethal, X-linked muscle-wasting disease caused by lack of the cytoskeletal protein dystrophin. There is currently no cure for DMD although various promising approaches are progressing through human clinical trials. By pharmacologically modulating the expression of the dystrophin-related protein utrophin, we have previously demonstrated in dystrophin-deficient mdx studies, daily SMT C1100 treatment significantly reduced muscle degeneration leading to impr...

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Authors: Guiraud, S; Squire, S; Edwards, B; Chen, H; Burns, D

• Publication status: Published
• Peer review status: Peer reviewed
• Publisher: Oxford University Press
• Journal: Human molecular genetics
• Volume: 24
• Issue: 15
• Pages: 4212-4224
• Publication date: 2015-05-01
• Acceptance date: 2015-04-27
• DOI: 10.1093/hmg/ddv154
• EISSN: 1460-2083
• ISSN: 0964-6906
• Pmid: 25935002
• Language: English
• Keywords: Muscular Dystrophy, Duchenne; tissue degeneration; Research Support, Non-U.S. Gov't; Muscular Dystrophy, Animal; skeletal muscles; Mice, Inbred mdx; administration; synapses; oral; Metabolism; Utrophin; muscle function; Pharmacotherapy; SBTMR; Humans; Gene Expression Regulation; cytoskeletal proteins; x-linked inheritance; Dystrophin; muscle fibers; Sarcolemma; Myocardium; Animals; tissue membrane; Muscles; muscular atrophy; Muscle Fibers, Skeletal; heart; fibrosis; research-article; Muscle Contraction; Mutation; necrosis; Journal Article; pathology; Mice; duchenne's muscular dystrophy