Therapeutic challenges in two adolescent male patients with Fabry disease and high antibody titres

Therapeutic challenges in two adolescent male patients with Fabry disease and high antibody titres

Enzyme replacement therapy (ERT) has been shown to stabilize certain aspects of Fabry disease (FD). However, in some patients on ERT, high antibody titres have been documented, with limited clinical improvement in systemic manifestations and often with significant adverse drug reactions. We present...

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Bibliographic Details
Main Authors: Aizeddin A. Mhanni, Christiane Auray-Blais, Michel Boutin, Alie Johnston, Kaye LeMoine, Jill Patterson, Johannes M.F.G. Aerts, Michael L. West, Cheryl Rockman-Greenberg
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Fabry disease
Agalsidase antibodies
Enzyme replacement therapy
Globotriaosylceramide (Gb3)
Globotriaosylsphingosine (Lyso-Gb3)
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426920300641

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http://www.sciencedirect.com/science/article/pii/S2214426920300641

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