Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb3) in Fabry Transgenic Mice and in the Plasma of Fabry Patients

PLoS ONE

Volumn 8, Issue 3, 2013, Pages

  Young Gqamana, Brandy ^a   Brignol, Nastry ^b   Chang, Hui Hwa ^b   Khanna, Richie ^b   Soska, Rebecca ^b   Fuller, Maria ^c   Sitaraman, Sheela A ^b   Germain, Dominique P ^d   Giugliani, Roberto ^e   Hughes, Derralynn A ^f   Mehta, Atul ^f   Nicholls, Kathy ^g   Boudes, Pol ^b   Lockhart, David J ^b   Valenzano, Kenneth J ^b   Benjamin, Elfrida R ^b  

^a UNIVERSITY OF CAPE TOWN   (South Africa)

^b Amicus Therapeutics   (United States)

^c WOMEN S AND CHILDREN S HOSPITAL   (Australia)

^d LABORATOIRE DES SCIENCES DU CLIMAT ET DE L ENVIRONNEMENT   (France)

^e FEDERAL UNIVERSITY OF RIO GRANDE DO SUL   (Brazil)

^f ROYAL FREE AND UNIVERSITY COLLEGE MEDICAL SCHOOL   (United Kingdom)

^g ROYAL MELBOURNE HOSPITAL   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE BETA; GLOBOTRIAOSYLCERAMIDE; GLOBOTRIAOSYLSPHINGOSINE; GLYCOSPHINGOLIPID; GR 181413A; LARAZOTIDE; MIGALASTAT; UNCLASSIFIED DRUG; 1 DEOXYNOJIRIMYCIN; ALPHA GALACTOSIDASE; CERAMIDE TRIHEXOSIDE; GLOBOTRIAOSYL LYSOSPHINGOLIPID; GLYCOLIPID; SPHINGOLIPID; SPHINGOSINE;

ACCURACY; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CALIBRATION; CLINICAL ARTICLE; CONTROLLED STUDY; ENZYME REPLACEMENT; FABRY DISEASE; HEART; HUMAN; KIDNEY; LIMIT OF QUANTITATION; LIQUID CHROMATOGRAPHY; MALE; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PHASE 2 CLINICAL TRIAL (TOPIC); SKIN; TANDEM MASS SPECTROMETRY; TRANSGENIC MOUSE; URINE; ANALOGS AND DERIVATIVES; ANIMAL; BLOOD; C57BL MOUSE; GENETICS; KNOCKOUT MOUSE; METABOLISM; MUTATION; ORAL DRUG ADMINISTRATION; REPRODUCIBILITY;

1-DEOXYNOJIRIMYCIN; ADMINISTRATION, ORAL; ALPHA-GALACTOSIDASE; ANIMALS; FABRY DISEASE; GLYCOLIPIDS; HUMANS; MALE; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; MUTATION; REPRODUCIBILITY OF RESULTS; SPHINGOLIPIDS; SPHINGOSINE; TRIHEXOSYLCERAMIDES;

EID: 84874602612     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0057631     Document Type: Article

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