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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Autores principales: 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
Lenguaje:Inglês
Publicado: The American Society for Biochemistry and Molecular Biology 2012
Materias:
Research Articles
Acceso en línea: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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