Enzyme-replacement therapy for metabolic storage disorders (Q33363790)

26 July 2017

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title

Enzyme-replacement therapy for metabolic storage disorders (English)

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enzyme replacement therapy

26 July 2017

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author name string

Roscoe O Brady

series ordinal

1

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PubMed ID

15556808

26 July 2017

Raphael Schiffmann

series ordinal

2

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26 July 2017

1 December 2004

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26 July 2017

1 reference

26 July 2017

3

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26 July 2017

752-756

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26 July 2017

12

1 reference

The Mainz Severity Score Index: a new instrument for quantifying the Anderson-Fabry disease phenotype, and the response of patients to enzyme replacement therapy.

26 July 2017

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

alpha-Glucosidase deficiency in generalized glycogenstorage disease (Pompe's disease).

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Cerebroside synthesis in Gaucher's disease

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzymatic Defect in Fabry's Disease

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

The defect in the Hurler and Scheie syndromes: deficiency of -L-iduronidase

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Hurler's syndrome, an -L-iduronidase deficiency

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Replacement Therapy for Inherited Enzyme Deficiency

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Replacement therapy for inherited enzyme deficiency. Use of purified glucocerebrosidase in Gaucher's disease.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Uptake and distribution of placental glucocerebrosidase in rat hepatic cells and effects of sequential deglycosylation

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Long-term safety and efficacy of enzyme replacement therapy for Fabry disease

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Ascorbate decreases Fabry cerebral hyperperfusion suggesting a reactive oxygen species abnormality: an arterial spin tagging study

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzyme replacement therapy in heterozygous females with Fabry disease: results of a phase IIIB study

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Improvement of cardiac function during enzyme replacement therapy in patients with Fabry disease: a prospective strain rate imaging study

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzyme replacement therapy improves peripheral nerve and sweat function in Fabry disease.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzyme replacement therapy improves function of C-, Adelta-, and Abeta-nerve fibers in Fabry neuropathy.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzyme-replacement therapy in mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Advances in the management of Anderson-Fabry disease: enzyme replacement therapy

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

Diagnosis of lysosomal storage disorders: current techniques and future directions.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2804%2900938-X

7 January 2021

10.1016/S1474-4422(04)00938-X

Identifiers

DOI

1 reference

26 July 2017

1 reference

PubMed ID

15556808