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- W2054503138 endingPage "584" @default.
- W2054503138 startingPage "567" @default.
- W2054503138 abstract "Enzyme replacement therapies substitute particular enzymes in individuals who have deficient or absent levels of enzymatic activity due to inherited defects. Fabry disease, a rare X-linked genetic disorder yet common lysosomal storage disease, is due to the impaired activity of α-galactosidase A. Currently, there are two enzyme preparations available for the treatment of Fabry disease: agalsidase alfa (Replagal®), manufactured by Shire, and agalsidase beta (Fabrazyme®), manufactured by Genzyme. Here, we review the molecular characteristics and intracellular trafficking of the endogenous and exogenous enzymes as well as the pharmacological properties of Replagal. We also summarize clinical experiences with Replagal and provide insight into its therapeutic value for the treatment of Fabry disease." @default.
- W2054503138 created "2016-06-24" @default.
- W2054503138 creator A5006254176 @default.
- W2054503138 creator A5035668327 @default.
- W2054503138 creator A5067876029 @default.
- W2054503138 date "2014-06-01" @default.
- W2054503138 modified "2023-10-18" @default.
- W2054503138 title "Agalsidase alfa for the treatment of fabry disease: a closer look" @default.
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