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Volume 2 (2015): Issue 1

Glad tidings for haemophilia B patients

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References

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  • 2. Rogaev EI, Grigorenko AP, Faskhutdinova G, Kittler EL, Moliaka YK. Genotype Analysis Identifies the Cause of the “Royal Disease”. Science 2009: 326; 817. doi: 10.1126/science.1180660.
  • 3. Quick AJ, Hussey CV. Hemophilia B (PTC deficiency, or Christmas disease). Arch Intern Med 1959; 103: 762–75. doi:10.1001/archinte.1959.00270050084014.
  • 4. Mannucci PM, Franchini M. Is haemophilia B less severe than haemophilia A? Haemophilia 2013; 19: 499-502. doi: 10.1111/hae.12133.
  • 5. Reijnen MJ, Sladek FM, Bertina RM, Reitsma PH. Disruption of a binding site for hepatocyte nuclear factor 4 results in hemophilia B Leyden. Proc Natl Acad Sci USA. 1992; 89: 6300-3.
  • 6. Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011; 365: 2357-65. doi: 10.1056/NEJMoa1108046.
  • 7. Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014; 371: 1994-2004. doi: 10.1056/NEJMoa1407309.
  • 8. Collins PW, Young G, Knobe K, et al. Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial. Blood First Edition Paper, prepublished online September 26, 2014; DOI 10.1182/blood-2014-05-573055

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