Property-based design of a glucosylceramide synthase inhibitor that reduces glucosylceramide in the brain

Synthesis inhibition is the basis for the treatment of type 1 Gaucher disease by the glucosylceramide synthase (GCS) inhibitor eliglustat tartrate. However, the extended use of eliglustat and related compounds for the treatment of glycosphingolipid storage diseases with CNS manifestations is limited...

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Detalhes bibliográficos
Main Authors: Larsen, Scott D., Wilson, Michael W., Abe, Akira, Shu, Liming, George, Christopher H., Kirchhoff, Paul, Showalter, H. D. Hollis, Xiang, Jianming, Keep, Richard F., Shayman, James A.
Formato: Artigo
Idioma:Inglês
Publicado em: The American Society for Biochemistry and Molecular Biology 2012
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3269155/
https://ncbi.nlm.nih.gov/pubmed/22058426
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1194/jlr.M021261
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