The Safety of Agalsidase Alfa Enzyme Replacement Therapy in Canadian Patients with Fabry Disease Following Implementation of a Bioreactor Process.

ABSTRACT:

Background and objective

Fabry disease, an X-linked lysosomal storage disorder characterized by absent or reduced alpha-galactosidase activity, is a lifelong disease that impairs patients' quality of life. Patients with Fabry disease have a considerably shortened lifespan, with mortality being mainly due to renal failure, cardiovascular disease, or cerebrovascular disease. Enzyme replacement therapy with agalsidase alfa has been shown to attenuate the renal, cardiovascular, and neuropathic disease progression associated with Fabry disease. The objective of this study was to investigate the safety of a new animal component-free version of agalsidase alfa.

Methods

A phase III/IV, open-label, single-arm, multicenter safety study was conducted in Canadian patients with Fabry dis

SUBMITTER: Khan A 

PROVIDER: S-EPMC8602602 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature