Disease Specific Therapies in Leukodystrophies and Leukoencephalopathies

The leukodystrophies are a heterogeneous, often progressive group of disorders manifesting a wide range of symptoms and complications. Most of these disorders have historically had no etiologic or disease specific therapeutic approaches. Recently, a greater understanding of the pathologic mechanisms...

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Bibliografiske detaljer
Udgivet i:Mol Genet Metab
Main Authors: Helman, Guy, Van Haren, Keith, Bonkowsky, Joshua L., Bernard, Genevieve, Pizzino, Amy, Braverman, Nancy, Suhr, Dean, Patterson, Marc C., Fatemi, S. Ali, Leonard, Jeff, van der Knaap, Marjo S, Back, Stephen A., Damiani, Stephen, Goldman, Steven A., Takanohashi, Asako, Petryniak, Magdalena, Rowitch, David, Messing, Albee, Wrabetz, Lawrence, Schiffmann, Raphael, Eichler, Florian, Escolar, Maria L., Vanderver, Adeline
Format: Artigo
Sprog:Inglês
Udgivet: 2015
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4390468/
https://ncbi.nlm.nih.gov/pubmed/25684057
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2015.01.014
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