J Intern Med. 2006 Jul;260(1):1-10.
A journey of hope: lessons learned from studies on rare diseases and orphan
drugs.
Wastfelt M, Fadeel B, Henter JI.
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This article gives interesting insights on the development of orphan drugs for rare disorders. It also contains a list of great websites.
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Philippe Campeau, MD
Resident in Medical Genetics at McGill University
OMMBID Blog Administrator