Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat (Q34079226)

30 July 2017

Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat (English)

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30 July 2017

phase II clinical trial

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eliglustat

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ChEBI release 2020-05-28

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Ravi S Kamath

1

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30 July 2017

Elena Lukina

2

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30 July 2017

Nora Watman

3

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30 July 2017

Marta Dragosky

4

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30 July 2017

Gregory M Pastores

5

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30 July 2017

Elsa Avila Arreguin

6

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30 July 2017

Hanna Rosenbaum

7

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30 July 2017

Ari Zimran

8

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30 July 2017

Rasha Aguzzi

9

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30 July 2017

Ana Cristina Puga

10

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30 July 2017

Andrea M Norfleet

11

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30 July 2017

M Judith Peterschmitt

12

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30 July 2017

Daniel I Rosenthal

13

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30 July 2017

10 May 2014

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30 July 2017

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30 July 2017

43

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1353-1360

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30 July 2017

10

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Creative Commons Attribution 4.0 International

30 July 2017

start time

10 May 2014

1 reference

April 2022 Public Data File from Crossref

copyright status

copyrighted

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exact match

https://scigraph.springernature.com/pub.10.1007/s00256-014-1891-9

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cites work

Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Therapeutic approaches to bone pathology in Gaucher disease: past, present and future

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Markers of bone turnover in Gaucher disease: modeling the evolution of bone disease

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Osteopenia in Gaucher disease develops early in life: response to imiglucerase enzyme therapy in children, adolescents and adults

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Glucocerebrosidase gene-deficient mouse recapitulates Gaucher disease displaying cellular and molecular dysregulation beyond the macrophage

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Eliglustat tartrate, an orally active glucocerebroside synthase inhibitor for the potential treatment of Gaucher disease and other lysosomal storage diseases.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

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

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Characterization of Gaucher disease bone marrow mesenchymal stromal cells reveals an altered inflammatory secretome

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Phenotype, diagnosis, and treatment of Gaucher's disease

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

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

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Improvement of bone disease by imiglucerase (Cerezyme) therapy in patients with skeletal manifestations of type 1 Gaucher disease: results of a 48-month longitudinal cohort study

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

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

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Evidence-based recommendations for monitoring bone disease and the response to enzyme replacement therapy in Gaucher patients

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Response of Gaucher bone disease to enzyme replacement therapy

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Imaging and quantifying skeletal involvement in Gaucher disease

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Gaucher disease: understanding the molecular pathogenesis of sphingolipidoses

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Skeletal complications of Gaucher disease

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Quantitative chemical shift imaging of vertebral bone marrow in patients with Gaucher disease

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Bone marrow response in treated patients with Gaucher disease: evaluation by T1-weighted magnetic resonance images and correlation with reduction in liver and spleen volume

5 July 2018

1 reference

Crossref

https://api.crossref.org/works/10.1007%2FS00256-014-1891-9

The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease.

21 January 2018

1 reference

Crossref

https://api.crossref.org/works/10.1007%2FS00256-014-1891-9

Spontaneous resolution of osteonecrosis of the femoral head

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4141971

Evaluation of Gaucher disease using magnetic resonance imaging

28 October 2018

1 reference

12 December 2020

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

1 reference

12 December 2020

Safety, tolerability, and pharmacokinetics of eliglustat tartrate (Genz-112638) after single doses, multiple doses, and food in healthy volunteers

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

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

1 reference

12 December 2020

Magnetic resonance imaging of bone marrow changes in Gaucher disease during enzyme replacement therapy: first German long-term results

1 reference

12 December 2020

10.1007/S00256-014-1891-9

Identifiers

DOI

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Dimensions Publication ID

30 July 2017

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30 July 2017

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