1. Global Genes. Rare diseases: fact and statistics. Statistics and Figures on Prevalence of Genetic and Rare Diseases Available at: https://globalgenes.org/rare-diseases-facts-statistics/ . Accessed 5 Dec 2017
2. Myshko D. Rare Diseases: Research, Development, and Commercialization. PharmaVOICE. 2015:15–21.
3. Pharmaceutical Manufacturers of America (PhRMA). Medicines in Development for Rare Diseases. A Report on Orphan Drugs in the Pipeline. 2016. Available at: http://phrma-docs.phrma.org/sites/default/files/pdf/medicines-in-development-report-rare-diseases.pdf . Accessed 5 Dec 2017.
4. US Department of Health and Human Services. Food and Drug Administration. Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER). Rare Diseases: Common Issues in Drug Development. Guidance for Industry. August 2015. Available at: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM458485.pdf . Accessed 5 Dec 2017.
5. Nijsten T, Bergstresser PR. Patient advocacy groups: let’s stick together. J Invest Dermatol. 2010;130:1757–9. [Editorial]