Time to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry.

ABSTRACT:

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 glycolipid deposits from endothelial cells within 6 months; clearance from other cell types required sustained treatment. We hypothesised that there might be a 'lag time' to clinical benefit after initiating agalsidase β treatment, and analysed the incidence of severe clinical events over time in patients receiving agalsidase β.

Methods

The incidence of severe clinical events (renal failure, cardiac events, stroke, death) was studied in 1044 adult patients (641 men, 403 women) enrol

SUBMITTER: Ortiz A 

PROVIDER: S-EPMC4941144 | biostudies-literature | 2016 Jul

REPOSITORIES: biostudies-literature