(Q33389419)

26 July 2017

title

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. (English)

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26 July 2017

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ChEBI release 2020-05-28

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ChEBI release 2020-05-28

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Elena Lukina

1

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26 July 2017

Nora Watman

2

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26 July 2017

Elsa Avila Arreguin

3

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26 July 2017

Maryam Banikazemi

4

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26 July 2017

Marta Dragosky

5

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26 July 2017

Marcelo Iastrebner

6

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26 July 2017

Hanna Rosenbaum

7

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26 July 2017

Mici Phillips

8

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26 July 2017

Gregory M Pastores

9

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26 July 2017

Daniel I Rosenthal

10

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26 July 2017

Mathilde Kaper

11

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26 July 2017

Tejdip Singh

12

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26 July 2017

Ana Cristina Puga

13

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26 July 2017

Peter L Bonate

14

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26 July 2017

M Judith Peterschmitt

15

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26 July 2017

language of work or name

English

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publication date

3 May 2010

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26 July 2017

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26 July 2017

volume

116

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26 July 2017

issue

6

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26 July 2017

page(s)

893-899

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Gaucher disease: a model disorder for biomarker discovery

26 July 2017

cites work

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2924227

Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2924227

Official Positions of the International Society for Clinical Densitometry and executive summary of the 2007 ISCD Position Development Conference

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2924227

Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2924227

Therapeutic goals in the treatment of Gaucher disease.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2924227

Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2924227

Marked elevation of plasma chitotriosidase activity. A novel hallmark of Gaucher disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2924227

Type I Gaucher disease, a glycosphingolipid storage disorder, is associated with insulin resistance

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2924227

Gaucher disease in Colombia: mutation identification and comparison to other Hispanic populations

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2924227

Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis.

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2924227

Safety, Tolerability, and Pharmacokinetics of Eliglustat Tartrate (Genz-112638) After Single Doses, Multiple Doses, and Food in Healthy Volunteers

31 October 2018

1 reference

12 December 2020

A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease

1 reference

12 December 2020

Enzyme replacement therapy for Gaucher disease: skeletal responses to macrophage-targeted glucocerebrosidase

1 reference

12 December 2020

Effect of enzyme replacement therapy with imiglucerase on BMD in type 1 Gaucher disease

1 reference

12 December 2020

Improved Inhibitors of Glucosylceramide Synthase

1 reference

12 December 2020

Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources

1 reference

12 December 2020

Analogs of ceramide that inhibit glucocerebroside synthetase in mouse brain

1 reference

12 December 2020

The fatigue severity scale. Application to patients with multiple sclerosis and systemic lupus erythematosus

1 reference

12 December 2020

Assessing individual outcomes during outpatient multidisciplinary chronic pain treatment by means of an augmented SF-36

1 reference

12 December 2020

Evaluation of Gaucher disease using magnetic resonance imaging

1 reference

12 December 2020

10.1182/BLOOD-2010-03-273151

Identifiers

DOI

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26 July 2017

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26 July 2017

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