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Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema.
Cohn, Danny M; Viney, Nicholas J; Fijen, Lauré M; Schneider, Eugene; Alexander, Veronica J; Xia, Shuting; Kaeser, Gwendolyn E; Nanavati, Charvi; Baker, Brenda F; Geary, Richard S; Levi, Marcel; Meijers, Joost C M; Stroes, Erik S G.
Afiliação
  • Cohn DM; 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 Pharma
  • Viney NJ; 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 Pharma
  • Fijen LM; 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 Pharma
  • Schneider E; 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 Pharma
  • Alexander VJ; 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 Pharma
  • Xia S; 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 Pharma
  • Kaeser GE; 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 Pharma
  • Nanavati C; 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 Pharma
  • Baker BF; 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 Pharma
  • Geary RS; 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 Pharma
  • Levi M; 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 Pharma
  • Meijers 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 Sciences, Amsterdam UMC, University of Amsterdam, and the Department of Molecular and Cellular Hemostasis, Sanquin Research (J.C.M.M.), Amsterdam; Ionis Pharma
  • Stroes 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, Amsterdam UMC, University of Amsterdam, and the Department of Molecular and Cellular Hemostasis, Sanquin Research (J.C.M.M.), Amsterdam; Ionis Pharma
N Engl J Med ; 383(13): 1242-1247, 2020 09 24.
Article em En | MEDLINE | ID: mdl-32877578
ABSTRACT
Hereditary 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.).
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Pré-Calicreína / Oligonucleotídeos Antissenso / Angioedemas Hereditários Tipo de estudo: Clinical_trials Limite: Adult / Female / Humans Idioma: En Revista: N Engl J Med Ano de publicação: 2020 Tipo de documento: Article
Texto completo
Imprimir
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PubMed Links
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Pré-Calicreína / Oligonucleotídeos Antissenso / Angioedemas Hereditários Tipo de estudo: Clinical_trials Limite: Adult / Female / Humans Idioma: En Revista: N Engl J Med Ano de publicação: 2020 Tipo de documento: Article