Time to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry

BACKGROUND: Agalsidase β is a form of enzyme replacement therapy for Fabry disease, a genetic disorder characterised by low α-galactosidase A activity, accumulation of glycosphingolipids and life-threatening cardiovascular, renal and cerebrovascular events. In clinical trials, agalsidase β cleared g...

詳細記述

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書誌詳細
出版年:J Med Genet
主要な著者: Ortiz, Alberto, Abiose, Ademola, Bichet, Daniel G, Cabrera, Gustavo, Charrow, Joel, Germain, Dominique P, Hopkin, Robert J, Jovanovic, Ana, Linhart, Aleš, Maruti, Sonia S, Mauer, Michael, Oliveira, João P, Patel, Manesh R, Politei, Juan, Waldek, Stephen, Wanner, Christoph, Yoo, Han-Wook, Warnock, David G
フォーマット: Artigo
言語:Inglês
出版事項: BMJ Publishing Group 2016
主題:
Therapeutics
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC4941144/
https://ncbi.nlm.nih.gov/pubmed/26993266
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/jmedgenet-2015-103486
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