Current Opinion in Hematology volume 20, issue 1, P41-47 2013 DOI: 10.1097/moh.0b013e32835a9148 View full text
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Derralynn A. Hughes, Gregory M. Pastores

Abstract: Improved understanding of biological and clinical risk factors for severe manifestations of Gaucher disease will allow rational assessment of patients and suggests potential nonsubstrate directed adjuvant strategies to consider in the management of this condition.