Effect of reduced agalsidase beta dosage in fabry patients: The Australian experience

Abstract

Background: In Australia, enzyme replacement therapy (ERT) for Fabry Disease (FD), both Agalsidase alfa (Replagal, Shire HGT) and beta (Fabrazyme, Genzyme), is funded and monitored through a specific government program. Agalsidase beta supply has been rationed by Genzyme since 2009 due to manufacturing issues. Consequently, the Australian Fabry Disease Advisory Committee has treated patients on Agalsidase beta at 50% of their usual dose from mid-2009, with a further reduction to 30% for some patients from late 2009. Aim: To determine the clinical effect of Agalsidase beta dose reduction in the Australian FD patient cohort. Methods: A questionnaire assessing FD symptoms was administered to 40..