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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
10:16

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease

Published on: December 20, 2017

Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible and safe

J Cox-Brinkman1, R G M Timmermans, F A Wijburg

  • 1Department of Pediatrics, Division of Metabolic Diseases, G8-205, Academic Medical Center, PO Box 22660, 1100 DD, Amsterdam, The Netherlands.

Journal of Inherited Metabolic Disease
|September 20, 2007
PubMed
Abstract

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