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Pharmacologic Management of Duchenne Muscular Dystrophy: Target Identification and Preclinical Trials

Duchenne muscular dystrophy (DMD) is an X-linked human disorder in which absence of the protein dystrophin causes degeneration of skeletal and cardiac muscle. For the sake of treatment development, over and above definitive genetic and cell-based therapies, there is considerable interest in drugs th...

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Главные авторы: Kornegay, Joe N., Spurney, Christopher F., Nghiem, Peter P., Brinkmeyer-Langford, Candice L., Hoffman, Eric P., Nagaraju, Kanneboyina
Формат: Artigo
Язык:Inglês
Опубликовано: Oxford University Press 2014
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Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4158345/
https://ncbi.nlm.nih.gov/pubmed/24936034
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/ilar/ilu011
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