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Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease

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-galactosidase A therapy. METHODS: The outcomes (seve...

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Detalles Bibliográficos
Autores principales:	Germain, Dominique P, Charrow, Joel, Desnick, Robert J, Guffon, Nathalie, Kempf, Judy, Lachmann, Robin H, Lemay, Roberta, Linthorst, Gabor E, Packman, Seymour, Scott, C Ronald, Waldek, Stephen, Warnock, David G, Weinreb, Neal J, Wilcox, William R
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BMJ Publishing Group 2015
Materias:	Therapeutics
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4413801/ https://www.ncbi.nlm.nih.gov/pubmed/25795794 http://dx.doi.org/10.1136/jmedgenet-2014-102797

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