Optimization of Eliglustat-Based Glucosylceramide Synthase Inhibitors as Substrate Reduction Therapy for Gaucher Disease Type 3

There remain no approved therapies for rare but devastating neuronopathic glyocosphingolipid storage diseases, such as Sandhoff, Tay-Sachs, and Gaucher disease type 3. We previously reported initial optimization of the scaffold of eliglustat, an approved therapy for the peripheral symptoms of Gauche...

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Detalhes bibliográficos
Publicado no:ACS Chem Neurosci
Main Authors: Wilson, Michael W., Shu, Liming, Hinkovska-Galcheva, Vania, Jin, Yafei, Rajeswaran, Walajapet, Abe, Akira, Zhao, Ting, Luo, Ruijuan, Wang, Lu, Wen, Bo, Liou, Benjamin, Fannin, Venette, Sun, Duxin, Sun, Ying, Shayman, James A., Larsen, Scott D.
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7919060/
https://ncbi.nlm.nih.gov/pubmed/33035424
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/acschemneuro.0c00558
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