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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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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Role:
Author
Journal:
Current gene therapy
Volume:
12
Issue:
3
Pages:
206-244
Publication date:
2012-06-05
DOI:
EISSN:
1875-5631
ISSN:
1566-5232
URN:
uuid:5bdbae0a-e0f6-4a5c-aa69-6d0c66d037bc
Source identifiers:
329116
Local pid:
pubs:329116

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