Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry
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- 作者：Robert J. Hopkina ; b ; rob.hopkin@cchmc.org" class="auth_mail" title="E-mail the corresponding author ; Gustavo Cabrerac ; gustavo.h.cabrera@hotmail.com" class="auth_mail" title="E-mail the corresponding author ; Joel Charrowd ; jcharrow@northwestern.edu" class="auth_mail" title="E-mail the corresponding author ; Roberta Lemaye ; Roberta.Lemay@genzyme.com" class="auth_mail" title="E-mail the corresponding author ; Ana Maria Martinsf ; martins.anamaria@uol.com.br" class="auth_mail" title="E-mail the corresponding author ; Michael Mauerg ; mauer002@umn.edu" class="auth_mail" title="E-mail the corresponding author ; Alberto Ortizh ; AOrtiz@fjd.es" class="auth_mail" title="E-mail the corresponding author ; Manesh R. Pateli ; patel017@mc.duke.edu" class="auth_mail" title="E-mail the corresponding author ; Katherine Simsj ; Sims@Helix.MGH.Harvard.edu" class="auth_mail" title="E-mail the corresponding author ; Stephen Waldekk ; stephen.waldek@sunderland.ac.uk" class="auth_mail" title="E-mail the corresponding author ; David G. Warnockl ; dwarnock@uab.edu" class="auth_mail" title="E-mail the corresponding author ; William R. Wilcoxm ; william.wilcox@emory.edu" class="auth_mail" title="E-mail the corresponding author
- 关键词：eGFR ; estimated glomerular filtration rate ; ERT ; enzyme replacement therapy ; ESRD ; end-stage renal disease ; GL-3 ; globotriaosylceramide ; IEC ; independent ethics committee ; IRB ; institutional review board ; LVH ; left ventricular hypertrophy ; UP/Cr ; urinary protein/creatinine ratio
- 刊名：Molecular Genetics and Metabolism
- 出版年：2016
- 出版时间：September-October 2016
- 年：2016
- 卷：119
- 期：1-2
- 页码：151-159
- 全文大小：734 K
文摘
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Risk factors for severe clinical events after enzyme replacement therapy initiation
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Risk factors include older age and advanced Fabry disease at treatment baseline.
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The observed patterns of residual risk may aid in clinical management of patients.