Orphan Drugs: Research and Reviews
Volume 4, 2014 - Issue
Open access
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Review
Orphan drugs in development for urea cycle disorders: current perspectives
Johannes Häberle1 Division of Metabolism and Children’s Research Center, University Children’s Hospital Zurich, Zurich, SwitzerlandCorrespondence[email protected]
& Shawn E McCandless2 Center for Human Genetics, University Hospitals Case Medical Center, and Department of Genetics and Genome Sciences, Case Western Reserve University, Cleveland, OH, USA
Pages 63-70
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Published online: 30 Sep 2014
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