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Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with fabry disease
Published Web Location
http://europepmc.org/articles/PMC4413801?pdf=renderNo data is associated with this publication.
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
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