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  • Title: Enzyme replacement in Gaucher disease.
    Author: Beutler E.
    Journal: PLoS Med; 2004 Nov; 1(2):e21. PubMed ID: 15578103.
    Abstract:
    The development of enzyme replacement therapy for Gaucher disease was a triumph of translational medicine. What were the key steps in its development? What are the controversies surrounding its use?
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