Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial (Q42733053)

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26 March 2020

title

Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28762527%20AND%20SRC:MED&resulttype=core&format=json

26 March 2020

main subject

phase III clinical trial

0 references

1 reference

1

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26 March 2020

author name string

Elena Lukina

2

1 reference

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26 March 2020

Hadhami Ben Turkia

3

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26 March 2020

Suma P Shankar

4

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26 March 2020

Hagit Baris

5

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26 March 2020

Marwan Ghosn

6

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26 March 2020

Atul Mehta

7

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26 March 2020

Seymour Packman

8

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26 March 2020

Gregory Pastores

9

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26 March 2020

Milan Petakov

10

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26 March 2020

Sarit Assouline

11

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26 March 2020

Manisha Balwani

12

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26 March 2020

Sumita Danda

13

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26 March 2020

Evgueniy Hadjiev

14

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26 March 2020

Andres Ortega

15

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26 March 2020

Sebastiaan J M Gaemers

16

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26 March 2020

Regina Tayag

17

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26 March 2020

M Judith Peterschmitt

18

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26 March 2020

language of work or name

1 reference

3 October 2017

1 reference

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American Journal of Hematology

26 March 2020

published in

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26 March 2020

volume

92

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26 March 2020

issue

11

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26 March 2020

page(s)

1170-1176

1 reference

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Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

26 March 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5656936

A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration

27 May 2018

1 reference

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Clonal Immunoglobulin against Lysolipids in the Origin of Myeloma

27 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5656936

Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial

27 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5656936

Type II NKT-TFH cells against Gaucher lipids regulate B-cell immunity and inflammation

27 May 2018

1 reference

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

27 May 2018

1 reference

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ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease

27 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5656936

Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response

27 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5656936

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

27 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5656936

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

27 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5656936

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

27 May 2018

1 reference

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Complement drives glucosylceramide accumulation and tissue inflammation in Gaucher disease.

27 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5656936

Glucosylsphingosine is a key biomarker of Gaucher disease

16 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5656936

Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial.

16 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5656936

Increased plasma macrophage inflammatory protein (MIP)-1alpha and MIP-1beta levels in type 1 Gaucher disease

16 August 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/28762527

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1002/AJH.24877

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28762527%20AND%20SRC:MED&resulttype=core&format=json

26 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28762527%20AND%20SRC:MED&resulttype=core&format=json

26 March 2020

PubMed publication ID

28762527

1 reference