[1. European Commission. Useful information on rare diseases from an EU perspective. Available at http://ec.europa.eu/health/ph_information/documents/ev20040705_rd05_en.pdf]Search in Google Scholar
[2. World Federation of Hemophilia. Annual Global Survey, 2013. Available at http://www1.wfh.org/publications/files/pdf-1591.pdf]Search in Google Scholar
[3. Peyvandi F, Palla R, Menegatti M, Mannucci PM. Introduction. Rare bleeding disorders: general aspects of clinical features, diagnosis, and management. Semin Thromb Hemost 2009; 35: 349-55.10.1055/s-0029-122575719598063]Search in Google Scholar
[4. Peyvandi F, Palla R, Menegatti M, et al. Coagulation factor activity and clinical bleeding severity in rare bleeding disorders: results from the European Network of Rare Bleeding Disorders. J Thromb Haemost 2012; 10(4): 615-21. doi: 10.1111/j.1538-7836.2012.04653.x.]Search in Google Scholar
[5. Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011;365(25): 2357-65. doi: 10.1056/NEJMoa1108046.]Search in Google Scholar
[6. Ward NJ, Buckley SM, Waddington SN, et al. Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood 2011;117(3):798-807. doi: 10.1182/blood-2010-05-282707.]Search in Google Scholar
[7. McIntosh J, Lenting PJ, Rosales C, et al. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood 2013; 121(17): 3335-44. doi: 10.1182/blood-2012-10-462200.]Search in Google Scholar