The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement (Q28182540)

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scientific article (publication date: 2003)

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English	The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement	scientific article (publication date: 2003)

Statements

scholarly article

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20 December 2019

1 reference

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

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20 December 2019

Gaucher's disease

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based on heuristic

inferred from title

2

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20 December 2019

Martin Hrebicek

14

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author name string

T M Cox

1

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20 December 2019

G Andria

3

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M Beck

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N Belmatoug

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B Bembi

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20 December 2019

R Chertkoff

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20 December 2019

S Vom Dahl

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20 December 2019

D Elstein

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20 December 2019

A Erikson

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M Giralt

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20 December 2019

R Heitner

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20 December 2019

C Hollak

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20 December 2019

S Lewis

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A Mehta

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G M Pastores

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20 December 2019

A Rolfs

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20 December 2019

M C Sa Miranda

19

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20 December 2019

A Zimran

20

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20 December 2019

Advisory Council to the European Working Group on Gaucher Disease

21

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language of work or name

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publication date

1 January 2003

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20 December 2019

Journal of Inherited Metabolic Disease

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20 December 2019

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513-526

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20 December 2019

6

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20 December 2019

Gaucher's disease: clinical features and natural history

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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The health-related quality of life of adults with Gaucher's disease receiving enzyme replacement therapy: results from a retrospective study.

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https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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Substrate reduction therapy for glycosphingolipid storage disorders

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https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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The natural course of Gaucher disease in The Netherlands: implications for monitoring of disease manifestations

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Quality of life assessment in adults with type 1 Gaucher disease.

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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High prevalence of low serum vitamin B12 in a multi-ethnic Israeli population

1 reference

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12 December 2020

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Withdrawal of enzyme replacement therapy in Gaucher's disease

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https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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Partial enzyme deficiencies: residual activities and the development of neurological disorders

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https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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N-butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis.

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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METABOLISM OF GLUCOCEREBROSIDES. II. EVIDENCE OF AN ENZYMATIC DEFICIENCY IN GAUCHER'S DISEASE.

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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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

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Glucocerebrosidase genotype of Gaucher patients in The Netherlands: limitations in prognostic value

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Effect of low-dose enzyme replacement therapy on bones in Gaucher disease patients with severe skeletal involvement

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Dixon quantitative chemical shift imaging is a sensitive tool for the evaluation of bone marrow responses to individualized doses of enzyme supplementation therapy in type 1 Gaucher disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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The safety and efficacy of combination N-butyl-deoxynojirimycin (SC-48334) and zidovudine in patients with HIV-1 infection and 200-500 CD4 cells/mm3.

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

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Gaucher's disease: genetic counselling and population screening.

1 reference

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12 December 2020

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Antibody response in patients with Gaucher disease after repeated infusion with macrophage-targeted glucocerebrosidase

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Gaucher disease. Clinical, laboratory, radiologic, and genetic features of 53 patients

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Clinical monitoring after cessation of enzyme replacement therapy in M. Gaucher

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Mutation analysis of the acid beta-glucosidase gene in a patient with type 3 Gaucher disease and neutralizing antibody to alglucerase

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Prevalence of lysosomal storage disorders

1 reference

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12 December 2020

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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://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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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/14605497

12 December 2020

based on heuristic

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Replacement therapy with imiglucerase for type 1 Gaucher's disease

1 reference

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12 December 2020

based on heuristic

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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/14605497

12 December 2020

based on heuristic

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Management of neuronopathic Gaucher disease: a European consensus.

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Low-dose enzyme replacement therapy for Gaucher's disease: effects of age, sex, genotype, and clinical features on response to treatment

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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The clinical course of treated and untreated Gaucher disease. A study of 45 patients

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Correlation of bone marrow response with hematological, biochemical, and visceral responses to enzyme replacement therapy of nonneuronopathic (type 1) Gaucher disease in 30 adult patients.

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Individualised low-dose alglucerase therapy for type 1 Gaucher's disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Interruption in enzyme replacement therapy for Gaucher disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Plasma and metabolic abnormalities in Gaucher's disease.

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Gaucher's disease: molecular, genetic and enzymological aspects

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Enzyme therapy in Gaucher disease type 1: dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Failure of alglucerase infused into Gaucher disease patients to localize in marrow macrophages.

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Report of the Spanish Gaucher's disease registry: clinical and genetic characteristics.

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

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Glucocerebrosidase (Gaucher disease).

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Low-dose N-butyldeoxynojirimycin (OGT 918) for type I Gaucher disease.

1 reference

https://pubmed.ncbi.nlm.nih.gov/14605497

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1023/A:1025902113005

2 references

Consolidated OpenCitations Corpus – April 2017

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Europe PubMed Central

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20 December 2019

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Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

2 references

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

Europe PubMed Central

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20 December 2019

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