Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy.
DMD is a devastatingly progressive muscle wasting disorder of childhood that significantly shortens life expectancy. Despite efforts to develop an effective therapy that dates back over a century, clinical interventions are still restricted to management of symptoms rather than a cure. The rationale...
| Main Authors: | , , |
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| 格式: | Journal article |
| 语言: | English |
| 出版: |
2012
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