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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Dettagli Bibliografici
Pubblicato in:J Med Genet
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: BMJ Publishing Group 2016
Soggetti:
Therapeutics
Accesso online: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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