Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease

Fabry disease, an X-linked glycosphingolipid storage disorder, is caused by the deficient activity of α-galactosidase A (α-Gal A). This results in the lysosomal accumulation in various cell types of its glycolipid substrates, including globotriaosylceramide (GL-3) and lysoglobotriaosylceramide (glob...

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Publicado no:Mol Med
Main Authors: Ashe, Karen M, Budman, Eva, Bangari, Dinesh S, Siegel, Craig S, Nietupski, Jennifer B, Wang, Bing, Desnick, Robert J, Scheule, Ronald K, Leonard, John P, Cheng, Seng H, Marshall, John
Formato: Artigo
Idioma:Inglês
Publicado em: ScholarOne 2015
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Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4559530/
https://ncbi.nlm.nih.gov/pubmed/25938659
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2119/molmed.2015.00088
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