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The impending medical revolution in haemophilia care: one patient’s view

   | 21 apr 2018
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Cita

1. Biggs R, Douglas AS, Macfarlane RC, et al. Christmas Disease. BMJ 1952; 2: 1378. doi: http://dx.doi.org/10.1136/bmj.2.4799.1378.Search in Google Scholar

2. Darby SC, Ewart DW, Giangrande PF, et al. Mortality before and after HIV infection in the complete UK population of haemophiliacs. Nature 1995; 377: 79Q82. doi:10.1038/377079a0Search in Google Scholar

3. Department of Health. Government response to Lord Archer’s Independent report on NHS supplied contaminated blood and blood products. London: Stationary Office; 20 May 2009Search in Google Scholar

4. Hütter G, Nowak D, Mossner M, et al. LongQterm control of HIV by CCR5 Delta32/Delta32 stemQcell transplantation. N Engl J Med 2009; 360R7S: 692Q8. doi: 10.1056/NEJMoa0802905.Search in Google Scholar

5. Roth DA, Kessler CM, Pasi KJ, et al; Recombinant Factor IX Study Group. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasmaQderived factor IX concentrates. Blood 2001; 98R13S: 3600Q6.10.1182/blood.V98.13.3600Open DOISearch in Google Scholar

6. Zaman SM, Hill FG, Palmer B, Millar CM, Bone A, Molesworth AM, et al. The risk of variant CreutzfeldtQJakob disease among UK patients with bleeding disorders, known to have received potentially contaminated plasma products. Haemophilia. 2011; 17R6S: 931Q7. doi: 10.1111/j.1365Q2516.2011.02508.x.Search in Google Scholar

7. Miguelino MG, Powell JS. Clinical utility and patient perspectives on the use of extended halfQlife rFIXFc in the management of hemophilia B. Patient Prefer Adherence 2014; 8: 1073Q83. doi: 10.2147/PPA.S54951.Search in Google Scholar

8. United States of America Food and Drug Administration. FDA approves first longQ acting recombinant coagulation Factor IX concentrate for patients with Hemophilia B. TOnlineU Available from: URL http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm391037.htmRLast accessed 12/12/2014S.Search in Google Scholar

9. Roosendaal G, Lafeber FP. Pathogenesis of haemophilic arthropathy. Haemophilia 2006; 12: RSuppl. 3S, 117V121.10.1111/j.1365-2516.2006.01268.x16684006Open DOISearch in Google Scholar

10. Cancio MI, Reiss UM, Nathwani AC, et al. Developments in the treatment of hemophilia B: focus on emerging gene therapy. Appl Clin Genet 2013; 6: 91Q101. doi: 10.2147/TACG.S31928.Search in Google Scholar

11. High KH, Nathwani A, Spencer T, Lillicrap D. Current status of haemophilia gene therapy. Haemophilia 2014; 20 Suppl 4: 43Q9. doi: 10.1111/hae.12411.Search in Google Scholar

12. Nathwani AC, Tuddenham EG, Rangarajan S, et al. AdenovirusQassociated virus vectorQmediated gene transfer in hemophilia B. N Engl J Med 2011; 365: 2357Q65. doi: 10.1056/NEJMoa1108046.Search in Google Scholar

13. Nathwani AC, Reiss UM, Tuddenham EG, et al. LongQterm safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014; 371: 1994Q2004. doi: 10.1056/NEJMoa1407309.Search in Google Scholar

14. High KA, Theodore E. Woodward Award. AAVQmediated gene transfer for haemophilia. Trans Am Clin Climatol Assoc 2003; 114: 337V352.Search in Google Scholar

15. World Federation of Hemophilia. TOnlineU. Annual Global Survey. Available from: URL http://www.wfh.org/en/resources/annualQglobalQsurvey,RLast accessed12/12/2014S.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