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Enzyme Replacement in Fabry Disease: The Essence Is in the Kidney.

Authors :
Schiffmann, Raphael
Source :
Annals of Internal Medicine. 1/16/2007, Vol. 146 Issue 2, p142-144. 3p.
Publication Year :
2007

Abstract

The article discusses the use of enzyme replacement therapy (ERT) to treat fabry disease. Fabry disease is a X-linked, single-gene defect caused by a deficiency of lysosomal α-galactosidase A resulting in failure to catabolize α-D-galactosyl glycolipid moieties. The 2 preparations of α-galactosidase A that are currently used for ERT are agalsidase alfa and agalsidase beta.

Subjects

Subjects :
*LYSOSOMAL storage diseases
*ENZYMES
*GALACTOSYLTRANSFERASES
*GLYCOLIPIDS
*MOIETIES (Chemistry)

Details

Language :
English
ISSN :
00034819
Volume :
146
Issue :
2
Database :
Academic Search Index
Journal :
Annals of Internal Medicine
Publication Type :
Academic Journal
Accession number :
23799395
Full Text :
https://doi.org/10.7326/0003-4819-146-2-200701160-00147
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