Successful desensitization with agalsidase alfa in 2 brothers with Fabry disease

Fabry disease is the only X-linked sphingolipidosis caused by a deficiency of the lysosomal enzyme alpha -galactosidase A. This deficiency results in the progressive accumulation of globotriaosylceramide (Gb3) and related glycosphingolipids in vascular endothelial cells, smooth muscle cells, and gan...

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Bibliographic Details
Published inJournal of investigational allergology & clinical immunology Vol. 23; no. 5; pp. 367 - 368
Main Authors Aydin, O, Kasapkara, C S, Celik, G E
Format Journal Article
LanguageEnglish
Published Spain 2013
Subjects
alpha-Galactosidase - adverse effects
alpha-Galactosidase - immunology
Desensitization, Immunologic - methods
Enzyme Replacement Therapy - adverse effects
Fabry Disease - drug therapy
Humans
Isoenzymes - adverse effects
Isoenzymes - immunology
Male
Middle Aged
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Summary:Fabry disease is the only X-linked sphingolipidosis caused by a deficiency of the lysosomal enzyme alpha -galactosidase A. This deficiency results in the progressive accumulation of globotriaosylceramide (Gb3) and related glycosphingolipids in vascular endothelial cells, smooth muscle cells, and ganglion cells, leading to ischemia and infarction especially in the kidney, heart, and brain. Renal and cardiovascular complications are the main cause of death in classic Fabry disease [1-3].
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ISSN:1018-9068