Efficacy of enzyme replacement therapy in Fabry disease.

Details

Serval ID
serval:BIB_A1B790CA0A88
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Institution
Title
Efficacy of enzyme replacement therapy in Fabry disease.
Journal
Current Medicinal Chemistry. Cardiovascular and Hematological Agents
Author(s)
Barbey F., Hayoz D., Widmer U., Burnier M.
ISSN
1568-0169 (Print)
ISSN-L
1568-0169
Publication state
Published
Issued date
10/2004
Volume
2
Number
4
Pages
277-286
Language
english
Notes
Publication types: Journal Article ; Review
Publication Status: ppublish
Abstract
Enzyme replacement therapy has recently been introduced to treat Fabry disease, a rare X-linked lysosomal storage disorder. The disease occurs due to deficient activity of alpha-galactosidase A, leading to progressive accumulation of globotriaosylceramide in multiple organs and tissues. Renal, cardiac and cerebrovascular manifestations of the disease result in premature death in both hemizygous males and heterozygous females. This paper outlines the clinical signs, symptoms and diagnosis of Fabry disease, and the development of the two available enzyme replacement therapies -- agalsidase alfa and agalsidase beta. Agalsidase alfa and agalsidase beta are produced in a human cell line and in Chinese hamster ovary cells, respectively, resulting in products with the same amino acid sequence as the native human enzyme, but with different patterns of glycosylation. Correct post-translational glycosylation is important in terms of the pharmacokinetics, biodistribution, clinical efficacy and tolerability of genetically engineered protein therapeutics. Differences in glycosylation, which may affect immunogenicity and mannose-6-phosphate receptor-mediated cellular internalisation of administered enzyme, possibly account for the differences in dosing, clinical effects and safety profiles reported for agalsidase alfa and agalsidase beta.
Keywords
Enzymes/administration & dosage, Enzymes/adverse effects, Fabry Disease/complications, Fabry Disease/therapy, Female, Humans, Isoenzymes/therapeutic use, Male, Treatment Outcome, alpha-Galactosidase/therapeutic use
Pubmed
Create date
17/01/2008 16:38
Last modification date
20/08/2019 15:07
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