10 Year Outcome of Enzyme Replacement Therapy with Agalsidase Beta in Patients with Fabry Disease. 

The aim of this study was to assess the clinical effect of α-galactosidase. Following supplementation, participants were assessed for severe clinical events, renal function and cardiac structure.  

This 10-year study documents the effectiveness of agalsidase beta (1 mg/kg/2 weeks) in patients with Fabry disease. Most patients remained alive and event-free. Patients who initiated treatment at a younger age and with less kidney involvement benefited the most from therapy.  

References:

Germain DP, Charrow J, Desnick RJ, Guffon N, Kempf J, Lachmann RH, Lemay R, Linthorst GE, Packman S, Scott CR, Waldek S, Warnock DG, Weinreb NJ, Wilcox WR. Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease. J Med Genet. 2015 May;52(5):353-8. doi: 10.1136/jmedgenet-2014-102797. Epub 2015 Mar 20. PMID: 25795794; PMCID: PMC4413801.