1. Globalgenes.org https://globalgenes.org/rare-diseases-facts-statistics/ 2016
2. Maisel , Y. A star is born: famous faces of rare disease - Progeria, Parkinson's, ALS, MS, SCID & more Primary-immunodeficiency.com http://www.primary-immunodeficiency.com/blog/a-star-is-born-famous-faces-of-rare-disease-fprogeria-parkinsons-als-ms-scid-more 2014
3. History of orphan drug regulation ---United States and beyond;Haffner;Clin. Pharmacol. Ther.,2016
4. US Food and Drug Administration Rare diseases: common issues in drug development guidance for industry 2015 http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM458485.pdf
5. Drugs against rare diseases: are the regulatory standards higher?;Gobburu;Clin. Pharmacol. Ther.,2016