Accesso libero

International databases open the door to improved care for rare bleeding disorders

INFORMAZIONI SU QUESTO ARTICOLO

Cita

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.pdfSearch in Google Scholar

2. World Federation of Hemophilia. Annual Global Survey, 2013. Available at http://www1.wfh.org/publications/files/pdf-1591.pdfSearch 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-0029-122575719598063Search 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

eISSN:
2055-3390
Lingua:
Inglese
Frequenza di pubblicazione:
Volume Open
Argomenti della rivista:
Medicine, Basic Medical Science, other, Clinical Medicine, Pharmacy, Pharmacology