Safety and Efficacy of Enzyme Replacement Therapy with Agalsidase Beta: An International, Open-label Study in Pediatric Patients with Fabry Disease

Article ID	Journal	Published Year	Pages	File Type
4167480	The Journal of Pediatrics	2008	9 Pages	PDF

Abstract

Agalsidase beta safely and effectively reduced the GL-3 accumulation in dermal endothelium already evident in children with Fabry disease. Early intervention may prevent irreversible end-organ damage from chronic GL-3 deposition.

Keywords

GFR αGal infusion-associated reaction GL-3 IAR OMIM SAE IgE IgG α-Galactosidase A enzyme replacement therapy enzyme-linked immunoadsorbent assay ECG IgE, immunoglobulin E immunoglobulin G ELISA Intravenous body mass index BMI adverse event Serious adverse event Glomerular filtration rate ERT Online Mendelian Inheritance in Man globotriaosylceramide

Related Topics

Health Sciences Medicine and Dentistry Perinatology, Pediatrics and Child Health

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Safety and Efficacy of Enzyme Replacement Therapy with Agalsidase Beta: An International, Open-label Study in Pediatric Patients with Fabry Disease

Authors

J. Edmond MB, ChB, Anna MD, PhD, Nathalie MD, PhD, Y. Howard MD, PhD, Michel MD, PhD, Ashok MBBS, Dominique P. MD, PhD,