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- Ernest Beutler.
- Department of Molecular and Experimental Medicine, Scripps Research Institute, La Jolla, California, USA. beutler@scripps.edu
- PLoS Med. 2004 Nov 1; 1 (2): e21e21.
AbstractThe 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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