Journal article
Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy.
- Abstract:
-
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 to develop effective therapies changed in 1986 with the discovery of the dystrophin gene. Since then extensive research into both the molecular basis and pathophysiology of DMD has paved the way n...
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- Publication status:
- Published
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- Publisher copy:
- 10.2174/156652312800840595
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Authors
Bibliographic Details
- Journal:
- Current gene therapy
- Volume:
- 12
- Issue:
- 3
- Pages:
- 206-244
- Publication date:
- 2012-06-01
- DOI:
- EISSN:
-
1875-5631
- ISSN:
-
1566-5232
- Source identifiers:
-
329116
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
pubs:329116
- UUID:
-
uuid:5bdbae0a-e0f6-4a5c-aa69-6d0c66d037bc
- Local pid:
- pubs:329116
- Deposit date:
- 2013-11-17
Terms of use
- Copyright date:
- 2012
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