Consequences of a global enzyme shortage of agalsidase beta in adult Dutch Fabry patients

Abstract Background Enzyme replacement therapy is currently the only approved therapy for Fabry disease. From June 2009 on, viral contamination of Genzyme's production facility resulted in a worldwide shortage of agalsidase beta leading to involuntary dose reductions (approved dose 1 mg/kg/eow, reduced dose 0.5 mg/kg/m), or switch to agalsidase alpha (administered dose 0.2 mg/kg/eow). An assessment report from the European Medicines Agency (EMA) raised serious concerns about an increase in adverse events at lower dosages of agalsidase beta. We determined the influence of the shortage on clinic... Mehr ...

Verfasser: Smid, Bouwien E
Rombach, Saskia M
Aerts, Johannes MFG
Kuiper, Symen
Mirzaian, Mina
Overkleeft, Hermen S
Poorthuis, Ben JHM
Hollak, Carla EM
Groener, Johanna EM
Linthorst, Gabor E
Dokumenttyp: Artikel
Erscheinungsdatum: 2011
Reihe/Periodikum: Orphanet Journal of Rare Diseases ; volume 6, issue 1 ; ISSN 1750-1172
Verlag/Hrsg.: Springer Science and Business Media LLC
Sprache: Englisch
Permalink: https://search.fid-benelux.de/Record/base-29464428
Datenquelle: BASE; Originalkatalog
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Link(s) : http://dx.doi.org/10.1186/1750-1172-6-69