Now showing items 1-3 of 3

    • AAV5-Factor VIII Gene Transfer in Severe Hemophilia A. 

      Rangarajan, S; Walsh, L; Lester, W; Perry, D; Madan, B; Laffan, M; Yu, H; Vettermann, C; Pierce, GF; Wong, WY (2017-12-28)
      BACKGROUND: Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, soft tissue, and the central nervous system. Although successful gene transfer has been reported in patients with hemophilia ...
    • Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A. 

      Pasi, KJ; Rangarajan, S; Mitchell, N; Lester, W; Symington, E; Madan, B; Laffan, M; Russell, CB; Li, M; Pierce, GF (Massachusetts Medical Society, 2020-01-02)
      BACKGROUND: Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years of follow-up after a single administration ...
    • Use of long term prophylaxis in London haemophilia centres 

      Austin, S; Chowdary, P; Kadam, A; Laffan, M; Dolan, G; Pasi, J (2016)