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...
| Hauptverfasser: | , , , , , , , , , , , , , |
|---|---|
| Format: | Journal article |
| Sprache: | English |
| Veröffentlicht: |
Public Library of Science
2011
|