Substrate reduction therapy for glycosphingolipid storage disorders (Q32066333)

6 July 2017

1 reference

title

Substrate reduction therapy for glycosphingolipid storage disorders (English)

1 reference

6 July 2017

Lachmann RH

1

1 reference

PubMed ID

11227045

6 July 2017

Platt FM

series ordinal

2

1 reference

PubMed ID

11227045

6 July 2017

language of work or name

English

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publication date

1 March 2001

1 reference

Expert Opinion on Investigational Drugs

6 July 2017

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6 July 2017

10

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6 July 2017

455-466

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6 July 2017

3

1 reference

Enzyme replacement therapy for Gaucher's disease

6 July 2017

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Gaucher's disease: clinical features and natural history

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Neuronopathic forms of Gaucher's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Clinical and biochemical outcome of marrow transplantation for Gaucher disease of the Norrbottnian type

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Gene therapy of lysosomal storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Human neural stem cells: isolation, expansion and transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Establishment and properties of neural stem cell clones: plasticity in vitro and in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

New therapeutic prospects for the glycosphingolipid lysosomal storage diseases.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Treatment of Gaucher disease with an enzyme inhibitor

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Reduction of globotriaosylceramide in Fabry disease mice by substrate deprivation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Modulation of cell-surface transferrin receptor by the imino sugar N-butyldeoxynojirimycin.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Tyrosinase and glycoprotein folding: roles of chaperones that recognize glycans

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Secretion of human hepatitis B virus is inhibited by the imino sugar N-butyldeoxynojirimycin

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Imino sugars inhibit the formation and secretion of bovine viral diarrhea virus, a pestivirus model of hepatitis C virus: implications for the development of broad spectrum anti-hepatitis virus agents

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Amino-sugar glycosidase inhibitors: versatile tools for glycobiologists

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Targeted disruption of the Hexa gene results in mice with biochemical and pathologic features of Tay-Sachs disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Neuropathology of mice with targeted disruption of Hexa gene, a model of Tay-Sachs disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Bone marrow transplantation prolongs life span and ameliorates neurologic manifestations in Sandhoff disease mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

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%2F13543784.10.3.455

Marked elevation of plasma chitotriosidase activity. A novel hallmark of Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Mice with disrupted GM2/GD2 synthase gene lack complex gangliosides but exhibit only subtle defects in their nervous system

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Mice lacking complex gangliosides develop Wallerian degeneration and myelination defects

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Autoantibodies associated with peripheral neuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Myelin-associated glycoprotein binding to gangliosides. Structural specificity and functional implications.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Withdrawal of enzyme replacement therapy in Gaucher's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

Improved Inhibitors of Glucosylceramide Synthase

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.10.3.455

10.1517/13543784.10.3.455

21 January 2018

Identifiers

DOI

1 reference

6 July 2017

1 reference

PubMed ID

11227045