Second-generation compound for the modulation of utrophin in the therapy of DMD
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...
| Главные авторы: | , , , , , , , , , , , , , |
|---|---|
| Формат: | Journal article |
| Язык: | English |
| Опубликовано: |
Oxford University Press
2015
|