• N. Engl. J. Med. · Sep 2020

    Case Reports Randomized Controlled Trial

    Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema.

    • Danny M Cohn, Nicholas J Viney, Lauré M Fijen, Eugene Schneider, Veronica J Alexander, Shuting Xia, Gwendolyn E Kaeser, Charvi Nanavati, Brenda F Baker, Richard S Geary, Marcel Levi, Meijers Joost C M JCM From the Departments of Vascular Medicine (D.M.C., L.M.F., E.S.G.S.) and Experimental Vascular Medicine (J.C.M.M.), Amsterdam Cardiovascular Science, and Stroes Erik S G ESG From the Departments of Vascular Medicine (D.M.C., L.M.F., E.S.G.S.) and Experimental Vascular Medicine (J.C.M.M.), Amsterdam Cardiovascular Sciences,.
    • From the Departments of Vascular Medicine (D.M.C., L.M.F., E.S.G.S.) and Experimental Vascular Medicine (J.C.M.M.), Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, and the Department of Molecular and Cellular Hemostasis, Sanquin Research (J.C.M.M.), Amsterdam; Ionis Pharmaceuticals, Carlsbad, CA (N.J.V., E.S., V.J.A., S.X., G.E.K., C.N., B.F.B., R.S.G.); and the Department of Medicine, University College London Hospitals NHS Foundation Trust, and the Cardiometabolic Programme, National Institute for Health Research University College London Hospitals and University College London Biomedical Research Centre, London (M.L.).
    • N. Engl. J. Med. 2020 Sep 24; 383 (13): 1242-1247.

    AbstractHereditary angioedema is characterized by recurrent and unpredictable episodes of subcutaneous and mucosal swelling that can be life threatening. IONIS-PKK-LRx is a ligand-conjugated antisense oligonucleotide designed for receptor-mediated delivery to hepatocytes. In a compassionate-use pilot study, two patients with severe bradykinin-mediated angioedema were initially administered weekly subcutaneous injections of the unconjugated parent drug, IONIS-PKKRx, for 12 to 16 weeks, after which they received IONIS-PKK-LRx at a dose of 80 mg every 3 to 4 weeks for 7 to 8 months. Treatment was accompanied by a reduction in the angioedema attack rate. (Funded by Amsterdam UMC.).Copyright © 2020 Massachusetts Medical Society.

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