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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Publicado en:J Med Genet
Autores principales: 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
Lenguaje:Inglês
Publicado: BMJ Publishing Group 2016
Materias:
Therapeutics
Acceso en línea: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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