Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients (Q34614418)

3 August 2017

title

Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients (English)

1 reference

3 August 2017

Dominique P Germain

8

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Derralynn A Hughes

object named as

Derralynn A Hughes

10

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Kathy Nicholls

object named as

Kathy Nicholls

12

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Roberto Giugliani

9

object named as

Roberto Giugliani

1 reference

3 August 2017

Brandy Young-Gqamana

1

1 reference

3 August 2017

Nastry Brignol

2

1 reference

3 August 2017

Hui-Hwa Chang

3

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3 August 2017

Richie Khanna

4

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3 August 2017

Rebecca Soska

5

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3 August 2017

Maria Fuller

6

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3 August 2017

Sheela A Sitaraman

7

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3 August 2017

Atul Mehta

11

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3 August 2017

Pol Boudes

13

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3 August 2017

David J Lockhart

14

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3 August 2017

Kenneth J Valenzano

15

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3 August 2017

Elfrida R Benjamin

16

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3 August 2017

language of work or name

English

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publication date

5 March 2013

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3 August 2017

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3 August 2017

volume

8

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3 August 2017

issue

3

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3 August 2017

page(s)

e57631

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Creative Commons Attribution 4.0 International

3 August 2017

copyright license

start time

5 March 2013

1 reference

April 2022 Public Data File from Crossref

copyright status

copyrighted

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cites work

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Long-term effect of antibodies against infused alpha-galactosidase A in Fabry disease on plasma and urinary (lyso)Gb3 reduction and treatment outcome

7 July 2018

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Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase A tissue uptake and improves substrate reduction in Fabry mice

7 July 2018

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Sex differences of urinary and kidney globotriaosylceramide and lyso-globotriaosylceramide in Fabry mice

7 July 2018

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7 July 2018

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Early therapeutic intervention in females with Fabry disease?

7 July 2018

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7 July 2018

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The role of ceramide trihexoside (globotriaosylceramide) in the diagnosis and follow-up of the efficacy of treatment of Fabry disease: a review of the literature

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Pharmacological chaperone corrects lysosomal storage in Fabry disease caused by trafficking-incompetent variants

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Is globotriaosylceramide a useful biomarker in Fabry disease?

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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21 January 2018

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21 January 2018

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How well does urinary lyso-Gb3 function as a biomarker in Fabry disease?

21 January 2018

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Tissue and plasma globotriaosylsphingosine could be a biomarker for assessing enzyme replacement therapy for Fabry disease

21 January 2018

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Quantification of the Fabry marker lysoGb3 in human plasma by tandem mass spectrometry.

21 January 2018

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LC-MS/MS analysis of plasma lyso-Gb3 in Fabry disease.

21 January 2018

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Urinary globotriaosylceramide excretion correlates with the genotype in children and adults with Fabry disease

21 January 2018

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Testosterone-responsive mouse kidney glycosphingolipids: developmental and inbred strain effects.

21 January 2018

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Long-term inhibition of glycosphingolipid accumulation in Fabry model mice by a single systemic injection of AAV1 vector in the neonatal period

21 January 2018

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21 January 2018

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Cell-Based Screening of Active-Site Specific Chaperone for the Treatment of Fabry Disease

21 January 2018

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12 December 2020

Enzymatic Defect in Fabry's Disease

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12 December 2020

Testosterone effects on the induction and urinary excretion of mouse kidney glycosphingolipids associated with lysosomes

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12 December 2020

Accelerated transport and maturation of lysosomal α–galactosidase A in Fabry lymphoblasts by an enzyme inhibitor

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12 December 2020

FABRY'S DISEASE: CLASSIFICATION AS A SPHINGOLIPIDOSIS AND PARTIAL CHARACTERIZATION OF A NOVEL GLYCOLIPID

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12 December 2020

Reduction of elevated plasma globotriaosylsphingosine in patients with classic Fabry disease following enzyme replacement therapy

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12 December 2020

Urinary globotriaosylsphingosine-related biomarkers for Fabry disease targeted by metabolomics

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12 December 2020

In vitro inhibition and intracellular enhancement of lysosomal α-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives

1 reference

12 December 2020

10.1371/JOURNAL.PONE.0057631

Identifiers

DOI

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3 August 2017

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3 August 2017

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