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1. Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with fabry disease

Author

Packman, Seymour, Germain, DP, Charrow, J, Desnick, RJ, Guffon, N, Kempf, J, Lachmann, RH, Lemay, R, Linthorst, GE, and Ronald, C

Abstract

Background: Fabry disease results from deficient α-galactosidase A activity and globotriaosylceramide accumulation causing renal insufficiency, strokes, hypertrophic cardiomyopathy and early demise. We assessed the 10-year outcome of recombinant α-galactos

Published
2015

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