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

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

By: Flora Peyvandi  
Open Access
|Apr 2018

References

  1. 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
  2. 2. World Federation of Hemophilia. Annual Global Survey, 2013. Available at http://www1.wfh.org/publications/files/pdf-1591.pdf
  3. 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
  4. 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.
  5. 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.
  6. 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.
  7. 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.
DOI: https://doi.org/10.17225/jhp00051 | Journal eISSN: 2055-3390
Language: English
Page range: 11 - 12
Published on: Apr 21, 2018
Published by: Haemnet Ltd
In partnership with: Paradigm Publishing Services
Publication frequency: 1 issue per year

© 2018 Flora Peyvandi, published by Haemnet Ltd
This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.