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Case Series

The Patient Experience of Gene Therapy for Hemophilia: Qualitative Interviews with Trial Patients

ORCID Icon & ORCID Icon
Pages 767-770 | Published online: 22 Apr 2020

References

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  • 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):2357–2365. doi:10.1056/NEJMoa1108046
  • Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014;371(21):1994–2004. doi:10.1056/NEJMoa1407309
  • George LA, Sullivan SK, Giermasz A, et al. Hemophilia B gene therapy with a high-specific-activity factor IX variant. N Engl J Med. 2017;377(23):2215–2227. doi:10.1056/NEJMoa1708538
  • Miesbach W, Meijer K, Coppens M, et al. Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B. Blood. 2018;131(9):1022–1031. doi:10.1182/blood-2017-09-804419
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