1. National Institutes of Health The Genetic and Rare Diseases Information Center (GARD) Fact Sheet 2010 http://report.nih.gov/nihfactsheets/Pdfs/TheGeneticAndRareDiseasesInformationCenter%28NHGRI%29.pdf
2. Rare diseases: the bane of modern society and the quest for cures;Azie;Clin. Pharmacol. Ther.,2012
3. Accelerating access to treatments for rare diseases;Dunoyer;Nat. Rev. Drug Discov.,2011
4. Food and Drug Administration Orphan Drug Act 2013 http://www.fda.gov/regulatoryinformation/legislation/federalfooddrugandcosmeticactfdcact/significantamendmentstothefdcact/orphandrugact/default.htm
5. Orphan products: an emerging trend in drug approvals;Cote;Nat. Rev. Drug Discov.,2010