Safety and Efficacy of Recombinant Human α-Galactosidase A Replacement Therapy in Fabry's Disease

M. Eng, Christine; Guffon, Nathalie; R. Wilcox, William; P. Germain, Dominique; Lee, Philip; Waldek, Steve; R. Caplan, Louis; E. Linthorst, Gabor; J. Desnick, Robert

doi:10.1056/nejm200107053450102

Public

Safety and Efficacy of Recombinant Human α-Galactosidase A Replacement Therapy in Fabry's Disease

Published in New England Journal of Medicine • Jul 5, 2001

Authors:

Christine M. Eng

Nathalie Guffon

William R. Wilcox

Abstract

BACKGROUND: Fabry's disease, lysosomal alpha-galactosidase A deficiency, results from the progressive accumulation of globotriaosylceramide and related glycosphingolipids. Affected patients have microvascular disease of the kidneys, heart, and brain. METHODS: We evaluated the safety and effectivenes...

Subject

Globotriaosylceramide

Medicine

Fabry disease

Research Assistant

AI chat, annotations, notes & similar papers

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!