Gaucher disease and the clinical experience with substrate reduction therapy (Q33348495)

26 July 2017

1 reference

title

Gaucher disease and the clinical experience with substrate reduction therapy (English)

1 reference

26 July 2017

1 reference

Ari Zimran

1

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PubMed ID

12803930

26 July 2017

Deborah Elstein

series ordinal

2

1 reference

PubMed ID

12803930

26 July 2017

language of work or name

English

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publication date

1 May 2003

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Philosophical Transactions of the Royal Society B

26 July 2017

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26 July 2017

358

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26 July 2017

1433

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26 July 2017

961-966

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Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease

26 July 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

Substrate reduction therapy in mouse models of the glycosphingolipidoses

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

Small-molecule therapeutics for the treatment of glycolipid lysosomal storage disorders

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

Enzyme replacement therapy: conception, chaos and culmination

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

Biochemistry of glycosphingolipid storage disorders: implications for therapeutic intervention

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

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=1693183

Agents for the treatment of glycosphingolipid storage disorders

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

Withdrawal of enzyme replacement therapy in Gaucher's 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=1693183

Enzyme therapy for Gaucher disease: the first 5 years.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

Gaucher's disease: molecular, genetic and enzymological aspects

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

Treatment of Gaucher disease with an enzyme inhibitor

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

The tolerability and pharmacokinetics of N-butyl-deoxynojirimycin in patients with advanced HIV disease (ACTG 100). The AIDS Clinical Trials Group (ACTG) of the National Institute of Allergy and Infectious Diseases

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

N-butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1693183

Low-dose N-butyldeoxynojirimycin (OGT 918) for type I 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=1693183

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=1693183

Pulmonary hypertension in two patients with type I Gaucher disease while on alglucerase therapy

31 October 2018

1 reference

12 December 2020

Echocardiographic assessment of pulmonary hypertension in Gaucher's disease

1 reference

12 December 2020

Lung involvement and enzyme replacement therapy in Gaucher's disease

1 reference

12 December 2020

Type I Gaucher disease in children with and without enzyme therapy

1 reference

12 December 2020

Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

1 reference

12 December 2020

Pulmonary hypertension developing after alglucerase therapy in two patients with type 1 Gaucher disease complicated by the hepatopulmonary syndrome

1 reference

12 December 2020

Identifiers

DOI

10.1098/RSTB.2003.1272

1 reference

26 July 2017

1 reference

26 July 2017

1 reference

PubMed ID

12803930