Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy (Q36932659)

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scientific article published on 25 March 2010

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English	Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy	scientific article published on 25 March 2010

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29 January 2020

Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy (English)

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29 January 2020

1

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29 January 2020

Diane P Copeland

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Diane P Copeland

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29 January 2020

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Kerry Anne McEachern

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29 January 2020

Wei-Lien Chuang

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29 January 2020

Elizabeth Hutto

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29 January 2020

Craig S Siegel

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29 January 2020

James A Shayman

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29 January 2020

Greg A Grabowski

7

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29 January 2020

Ronald K Scheule

8

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29 January 2020

Seng H Cheng

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29 January 2020

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25 March 2010

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29 January 2020

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9

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Journal of Inherited Metabolic Disease

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29 January 2020

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29 January 2020

3

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29 January 2020

281-289

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29 January 2020

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

29 September 2017

Chemical and biological approaches synergize to ameliorate protein-folding diseases.

1 reference

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29 September 2017

Gaucher disease: unmet treatment needs

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

29 September 2017

Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.

1 reference

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29 September 2017

Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system

1 reference

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29 September 2017

Pharmacological inhibition of glucosylceramide synthase enhances insulin sensitivity

1 reference

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29 September 2017

Effect of mannose chain length on targeting of glucocerebrosidase for enzyme replacement therapy of Gaucher disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

29 September 2017

Isofagomine- and 2,5-anhydro-2,5-imino-D-glucitol-based glucocerebrosidase pharmacological chaperones for Gaucher disease intervention.

1 reference

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29 September 2017

Structure of acid beta-glucosidase with pharmacological chaperone provides insight into Gaucher disease

1 reference

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29 September 2017

The long-term international safety experience of imiglucerase therapy for Gaucher disease

1 reference

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29 September 2017

Effective cell and gene therapy in a murine model of Gaucher disease

1 reference

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29 September 2017

The iminosugar isofagomine increases the activity of N370S mutant acid beta-glucosidase in Gaucher fibroblasts by several mechanisms

1 reference

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29 September 2017

Limitations of enzyme replacement therapy: current and future

1 reference

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29 September 2017

An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment

1 reference

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29 September 2017

Storage solutions: treating lysosomal disorders of the brain

1 reference

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29 September 2017

New directions in the treatment of Gaucher disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

29 September 2017

Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

29 September 2017

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

1 reference

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29 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

29 September 2017

Sphingolipid activator proteins are required for epidermal permeability barrier formation

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29 September 2017

Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin

1 reference

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29 September 2017

Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher's disease

1 reference

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29 September 2017

Kifunensine, a potent inhibitor of the glycoprotein processing mannosidase I.

1 reference

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29 September 2017

Viable mouse models of acid beta-glucosidase deficiency: the defect in Gaucher disease

1 reference

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27 June 2018

Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme.

1 reference

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4 September 2018

Enzyme replacement therapy for mild patients with Gaucher disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

4 September 2018

A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase

1 reference

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4 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

4 September 2018

Short-term effect of miglustat in every day clinical use in treatment-naïve or previously treated patients with type 1 Gaucher's disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

4 September 2018

AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

4 September 2018

Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3683842

4 September 2018

Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation.

1 reference

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4 September 2018

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/20336375

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Improved Inhibitors of Glucosylceramide Synthase

1 reference

https://pubmed.ncbi.nlm.nih.gov/20336375

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Superior effects of high-dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels: a 2-center retrospective analysis

1 reference

https://pubmed.ncbi.nlm.nih.gov/20336375

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Inhibiting glycosphingolipid synthesis improves glycemic control and insulin sensitivity in animal models of type 2 diabetes

1 reference

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/20336375

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Goal-oriented therapy with miglustat in Gaucher disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/20336375

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

A randomized trial comparing the efficacy and safety of imiglucerase (Cerezyme) infusions every 4 weeks versus every 2 weeks in the maintenance therapy of adult patients with Gaucher disease type 1

1 reference

https://pubmed.ncbi.nlm.nih.gov/20336375

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Preparation of the active isomer of 1-phenyl-2-decanoylamino-3-morpholino-1-propanol, inhibitor of murine glucocerebroside synthetase

1 reference

https://pubmed.ncbi.nlm.nih.gov/20336375

12 December 2020

based on heuristic

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Identifiers

10.1007/S10545-010-9072-Z

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Europe PubMed Central

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29 January 2020

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20336375 AND SRC:MED&resulttype=core&format=json

29 January 2020

PubMed publication ID

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20336375 AND SRC:MED&resulttype=core&format=json

29 January 2020

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