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Pharmacological therapeutics targeting the secondary defects and downstream pathology of Duchenne muscular dystrophy

INTRODUCTION: Since the identification of the dystrophin gene in 1986, a cure for Duchenne muscular dystrophy (DMD) has yet to be discovered. Presently, there are a number of genetic-based therapies in development aimed at restoration and/or repair of the primary defect. However, growing understandi...

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Detalhes bibliográficos
Publicado no:Expert Opin Orphan Drugs
Main Authors: Spinazzola, Janelle M., Kunkel, Louis M.
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5487007/
https://ncbi.nlm.nih.gov/pubmed/28670506
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/21678707.2016.1240613
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