Plasma glucosylsphingosine correlations with baseline disease burden and response to eliglustat in two clinical trials of previously untreated adults with Gaucher disease type 1-Reference-Cited by-同舟云学术

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Plasma glucosylsphingosine correlations with baseline disease burden and response to eliglustat in two clinical trials of previously untreated adults with Gaucher disease type 1

Published:2023-03 Issue:3 Volume:138 Page:107527
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Peterschmitt M. Judith,Foster Meredith C.,Ji Allena J.,Zajdel Marianne B.,Cox Gerald F.

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference39 articles.

1. Gaucher disease;Grabowski,2013

2. Lysosomal glycosphingolipid catabolism by acid ceramidase: formation of glycosphingoid bases during deficiency of glycosidases;Ferraz;FEBS Lett.,2016

3. Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response;Dekker;Blood,2011

4. Plasma glucosylceramide and ceramide in type 1 Gaucher disease patients: correlations with disease severity and response to therapeutic intervention;Groener;Biochim. Biophys. Acta,2008

5. Plasma chitotriosidase activity versus plasma glucosylsphingosine in wide spectrum of Gaucher disease phenotypes - a statistical insight;Tylki-Szymanska;Mol. Genet. Metab.,2018

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