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

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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Bibliographic Details
Main Authors: Scott D. Larsen, Michael W. Wilson, Akira Abe, Liming Shu, Christopher H. George, Paul Kirchhoff, H. D. Hollis Showalter, Jianming Xiang, Richard F. Keep, James A. Shayman
Format: Article
Language:English
Published: Elsevier 2012-02-01
Series:Journal of Lipid Research
Subjects:
Gaucher disease
blood-brain barrier
glycosphingolipid
synthesis inhibition therapy
eliglustat tartrate
Online Access:http://www.sciencedirect.com/science/article/pii/S0022227520407862

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http://www.sciencedirect.com/science/article/pii/S0022227520407862

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