(Q35056076)

6 August 2017

1 reference

title

Substrate reduction therapy: miglustat as a remedy for symptomatic patients with Gaucher disease type 1. (English)

1 reference

6 August 2017

1 reference

Gregory M Pastores

1

1 reference

PubMed ID

12556220

6 August 2017

Natalie L Barnett

series ordinal

2

1 reference

PubMed ID

12556220

6 August 2017

language of work or name

English

0 references

publication date

1 February 2003

1 reference

Expert Opinion on Investigational Drugs

6 August 2017

1 reference

6 August 2017

12

1 reference

6 August 2017

2

1 reference

6 August 2017

273-281

1 reference

Agents for the treatment of glycosphingolipid storage disorders

6 August 2017

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

New therapeutic prospects for the glycosphingolipid lysosomal storage diseases.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

The glycosphingolipidoses-from disease to basic principles of metabolism

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Substrate reduction therapy for glycosphingolipid storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Chemical chaperones increase the cellular activity of N370S beta -glucosidase: a therapeutic strategy for Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Animal model of Gaucher's disease from targeted disruption of the mouse glucocerebrosidase gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Systemic inflammation in glucocerebrosidase-deficient mice with minimal glucosylceramide storage

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

A genetic model of substrate deprivation therapy for a glycosphingolipid storage disorder

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

A mouse B16 melanoma mutant deficient in glycolipids

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Pathogenesis of lysosomal storage disorders as illustrated by Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Glucosylceramide modulates membrane traffic along the endocytic pathway

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Glucosylsphingosine accumulation in tissues from patients with Gaucher disease: correlation with phenotype and genotype

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Cessation of enzyme replacement therapy in Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Biochemical Basis of Late-Onset Neurolipidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

Molecular pathophysiology in Tay-Sachs and Sandhoff diseases as revealed by gene expression profiling.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.12.2.273

10.1517/13543784.12.2.273

21 January 2018

Identifiers

DOI

1 reference

6 August 2017

1 reference

PubMed ID

12556220