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Agalsidase beta is a recombinant human alpha-galactosidase indicated to treat Fabry disease, a genetic deficiency in the enzyme leading to buildup of globotriaosylceramide.
Agalsidase beta is a recombinant human alpha-galactosidase indicated to treat Fabry disease, a genetic deficiency in the enzyme leading to buildup of globotriaosylceramide.
α-galactosidase A is uptaken by cells via the mannose 6 phosphate receptor. Agalsidase beta hydrolyzes globotriaosylceramide and other glycosphingolipids that would normally be hydrolyzed by endogenous α-galactosidase A. Preventing the accumulation of glycosphingolipids prevents or reduces the severity of manifestations of Fabry disease such as renal failure, cardiomyopathy, or cerebrovascular events.
Agalsidase beta is indicated in the treatment of Fabry disease.
1 associated conditions