Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasm...
Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients
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Author / Creator
Young-Gqamana, Brandy , Brignol, Nastry , Chang, Hui-Hwa , Khanna, Richie , Soska, Rebecca , Fuller, Maria , Sitaraman, Sheela A , Germain, Dominique P , Giugliani, Roberto , Hughes, Derralynn A , Mehta, Atul , Nicholls, Kathy , Boudes, Pol , Lockhart, David J , Valenzano, Kenneth J and Benjamin, Elfrida R
Publisher
United States: Public Library of Science
Journal title
Language
English
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Publisher
United States: Public Library of Science
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Scope and Contents
Contents
Fabry disease (FD) results from mutations in the gene (GLA) that encodes the lysosomal enzyme α-galactosidase A (α-Gal A), and involves pathological accumulation of globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb3). Migalastat hydrochloride (GR181413A) is a pharmacological chaperone that selectively binds, stabilizes, and increas...
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Full title
Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients
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Record Identifier
TN_cdi_plos_journals_1330880732
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_1330880732
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ISSN
1932-6203
E-ISSN
1932-6203
DOI
10.1371/journal.pone.0057631
