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Recommendations to enable drug development for inherited neuropathies: Charcot-Marie-Tooth and Giant Axonal Neuropathy

Approximately 1 in 2500 Americans suffer from Charcot-Marie-Tooth (CMT) disease. The underlying disease mechanisms are unique in most forms of CMT, with many point mutations on various genes causing a toxic accumulation of misfolded proteins. Symptoms of the disease often present within the first tw...

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Detalles Bibliográficos
Main Authors: Sames, Lori, Moore, Allison, Arnold, Renee, Ekins, Sean
Formato: Artigo
Idioma:Inglês
Publicado: F1000Research 2014
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4023663/
https://ncbi.nlm.nih.gov/pubmed/24860645
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.12688/f1000research.3751.2
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