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Enzyme Replacement Therapy in Fabry Disease

20011,301 citationsJournal Article

Authors

Raphael Schiffmann · National Institute of Neurological Disorders and Stroke

Jeffrey B. Kopp

Howard A. Austin

Sharda G. Sabnis

Roscoe O. Brady

Abstract

Intravenous infusions of alpha-gal A are safe and have widespread therapeutic efficacy in Fabry disease.

Topics & Keywords

Lysosomal Storage Disorders Research Botulinum Toxin and Related Neurological Disorders Glycogen Storage Diseases and Myoclonus

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: JAMA

Volume 285, Issue 21, pp. 2743-2743

DOI: 10.1001/jama.285.21.2743

Field-Weighted Citation Impact: 33.13

Enzyme Replacement Therapy in Fabry Disease | Researchclopedia