Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy.

@article{Pasi2017TargetingOA,
  title={Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy.},
  author={K. John Pasi and S Rangarajan and Pencho Georgiev and Tim G Mant and Michael Desmond Creagh and Toshko J. Lissitchkov and David Bevan and Steve Austin and Charles R. M. Hay and Inga Hegemann and Rashid Saeed Kazmi and Pratima Chowdary and Liana Gercheva-Kyuchukova and Vasily Mamonov and Margarita Timofeeva and Chang-Heok Soh and Pushkal P. Garg and Akshay K. Vaishnaw and Akin Akinc and Benny S\orensen and Margaret V Ragni},
  journal={The New England journal of medicine},
  year={2017},
  volume={377 9},
  pages={
          819-828
        }
}
BACKGROUND Current hemophilia treatment involves frequent intravenous infusions of clotting factors, which is associated with variable hemostatic protection, a high treatment burden, and a risk of the development of inhibitory alloantibodies. Fitusiran, an investigational RNA interference (RNAi) therapy that targets antithrombin (encoded by SERPINC1), is in development to address these and other limitations. METHODS In this phase 1 dose-escalation study, we enrolled 4 healthy volunteers and… CONTINUE READING

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