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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Veröffentlicht in:J Med Genet
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: BMJ Publishing Group 2015
Schlagworte:
Therapeutics
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4413801/
https://ncbi.nlm.nih.gov/pubmed/25795794
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/jmedgenet-2014-102797
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