(Q36739788)

16 August 2017

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title

Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders (English)

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16 August 2017

Terry D Butters

1

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16 August 2017

language of work or name

English

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publication date

1 March 2007

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Expert Opinion on Pharmacotherapy

16 August 2017

published in

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16 August 2017

volume

8

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16 August 2017

issue

4

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16 August 2017

page(s)

427-435

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Lysosomal storage disorders

16 August 2017

cites work

1 reference

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Lysosomal storage diseases: natural history and ethical and economic aspects

21 January 2018

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Bicistronic lentiviral vector corrects beta-hexosaminidase deficiency in transduced and cross-corrected human Sandhoff fibroblasts.

21 January 2018

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Reversion of the biochemical defects in murine embryonic Sandhoff neurons using a bicistronic lentiviral vector encoding hexosaminidase alpha and beta

21 January 2018

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Retrovirus-mediated enzymatic correction of Tay-Sachs defect in transduced and non-transduced cells

21 January 2018

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beta-hexosaminidase lentiviral vectors: transfer into the CNS via systemic administration.

21 January 2018

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Effective gene therapy in an authentic model of Tay-Sachs-related diseases

21 January 2018

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

21 January 2018

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A direct gene transfer strategy via brain internal capsule reverses the biochemical defect in Tay-Sachs disease

21 January 2018

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

21 January 2018

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Gene therapy for lysosomal storage diseases.

21 January 2018

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Imino sugar inhibitors for treating the lysosomal glycosphingolipidoses

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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Gaucher disease and parkinsonism

21 January 2018

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

21 January 2018

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Gaucher disease and the clinical experience with substrate reduction therapy

21 January 2018

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The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease

21 January 2018

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

21 January 2018

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Cellular effects of deoxynojirimycin analogues: uptake, retention and inhibition of glycosphingolipid biosynthesis

21 January 2018

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X-ray structure of human acid-beta-glucosidase covalently bound to conduritol-B-epoxide. Implications for Gaucher disease

21 January 2018

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Miglustat (NB-DNJ) works as a chaperone for mutated acid beta-glucosidase in cells transfected with several Gaucher disease mutations

21 January 2018

1 reference

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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%2F14656566.8.4.427

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

21 January 2018

1 reference

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A synthetic chaperone corrects the trafficking defect and disease phenotype in a protein misfolding disorder

21 January 2018

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Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis

21 January 2018

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Beta-galactosidase deficiency: an approach to chaperone therapy

21 January 2018

1 reference

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Galactonojirimycin derivatives restore mutant human beta-galactosidase activities expressed in fibroblasts from enzyme-deficient knockout mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.8.4.427

Pharmacological enhancement of beta-hexosaminidase activity in fibroblasts from adult Tay-Sachs and Sandhoff Patients.

21 January 2018

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A contradictory treatment for lysosomal storage disorders: inhibitors enhance mutant enzyme activity

21 January 2018

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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%2F14656566.8.4.427

NSAIDs increase survival in the Sandhoff disease mouse: synergy with N-butyldeoxynojirimycin

21 January 2018

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Prevalence of lysosomal storage disorders

21 January 2018

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

21 January 2018

1 reference

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Guidance on the use of miglustat for treating patients with type 1 Gaucher disease

21 January 2018

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

21 January 2018

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Limitations of enzyme replacement therapy: current and future

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.8.4.427

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%2F14656566.8.4.427

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.8.4.427

Substrate reduction therapy in the infantile form of Tay-Sachs disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.8.4.427

Treatment with miglustat reverses the lipid-trafficking defect in Niemann-Pick disease type C.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.8.4.427

21 January 2018

Identifiers

DOI

10.1517/14656566.8.4.427

1 reference

16 August 2017

1 reference