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...

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Detalhes bibliográficos
Publicado no:J Med Genet
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: BMJ Publishing Group 2016
Assuntos:
Therapeutics
Acesso em linha: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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