Fabry disease: recent advances in enzyme replacement therapy (Q34974879)

5 August 2017

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title

Fabry disease: recent advances in enzyme replacement therapy (English)

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enzyme replacement therapy

5 August 2017

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Dominique P Germain

1

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PubMed ID

12387706

5 August 2017

language of work or name

English

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publication date

1 October 2002

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Expert Opinion on Investigational Drugs

5 August 2017

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5 August 2017

11

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5 August 2017

10

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5 August 2017

1467-1476

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Fabry's Disease: Alpha-Galactosidase Deficiency

5 August 2017

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Ein Beitrag zur Kenntniss der Purpura haemorrhagica nodularis (Purpura papulosa haemorrhagica Hebrae)

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

A CASE OF “ANGEIO-KERATOMA.”

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Enhanced endothelium-dependent vasodilation in Fabry disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Non-invasive evaluation of arterial involvement in patients affected with Fabry disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

An Atypical Variant of Fabry's Disease with Manifestations Confined to the Myocardium

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Natural history of Fabry renal disease: influence of alpha-galactosidase A activity and genetic mutations on clinical course

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Prevalence of Anderson-Fabry disease in male patients with late onset hypertrophic cardiomyopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Cerebrovascular complications of Fabry's disease

21 January 2018

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https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Prevalence of lysosomal storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Expression of the human alpha-galactosidase A in Escherichia coli K-12.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Expression and characterization of glycosylated and catalytically active recombinant human alpha-galactosidase A produced in Pichia pastoris

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Insect cells as hosts for the expression of recombinant glycoproteins.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Cell-type-specific and site-specific N-glycosylation of type I and type II human tissue plasminogen activator

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Enzyme Replacement in Fabry's Disease, an Inborn Error of Metabolism

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Enzyme therapy in Fabry disease: differential in vivo plasma clearance and metabolic effectiveness of plasma and splenic alpha-galactosidase A isozymes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

alpha-Galactosidase A deficient mice: a model of Fabry disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Aging accentuates and bone marrow transplantation ameliorates metabolic defects in Fabry disease mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

A phase 1/2 clinical trial of enzyme replacement in fabry disease: pharmacokinetic, substrate clearance, and safety studies

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Regional cerebral hyperperfusion and nitric oxide pathway dysregulation in Fabry disease: reversal by enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Patients with Fabry disease on dialysis in the United States

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

Elevated cerebral blood flow velocities in Fabry disease with reversal after enzyme replacement.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.11.10.1467

10.1517/13543784.11.10.1467

21 January 2018

Identifiers

DOI

1 reference

5 August 2017

1 reference

PubMed ID

12387706