Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
10833456 | Molecular Genetics and Metabolism | 2012 | 9 Pages |
Abstract
⺠Anderson-Fabry disease is an X-linked lysosomal storage disorder resulting from the deficiency of the hydrolytic enzyme alpha galactosidase A. ⺠The current causal treatment for Fabry disease is the enzyme replacement therapy with two different products, Replagal (agalsidase alfa) and Fabrazyme (agalsidase beta) that have been commercially available in Europe for almost 10 years. ⺠This article reviews the published evidence of the clinical efficacy of the two available enzyme preparations. ⺠This review also focuses on the novel treatments and therapeutic options that are currenty available in patients with Anderson-Fabry disease.
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Authors
Antonio Pisani, Bianca Visciano, Graciana Diez Roux, Massimo Sabbatini, Caterina Porto, Giancarlo Parenti, Massimo Imbriaco,