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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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Cost-effectiveness of enzyme replacement therapy for Fabry disease

Saskia M Rombach1, Carla E M Hollak, Gabor E Linthorst

  • 1Department of Internal Medicine, Division of Endocrinology and Metabolism, Academic Medical Centre, PO Box 22660, Amsterdam, DD 1100, The Netherlands.

Orphanet Journal of Rare Diseases
|February 21, 2013
PubMed
Abstract

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