TY - JOUR
T1 - Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema
AU - Cohn, Danny M.
AU - Viney, Nicholas J.
AU - Fijen, Lauré M.
AU - Schneider, Eugene
AU - Alexander, Veronica J.
AU - Xia, Shuting
AU - Kaeser, Gwendolyn E.
AU - Nanavati, Charvi
AU - Baker, Brenda F.
AU - Geary, Richard S.
AU - Levi, Marcel
AU - Meijers, Joost C. M.
AU - Stroes, Erik S. G.
PY - 2020/9/24
Y1 - 2020/9/24
N2 - 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.).
AB - 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.).
UR - http://www.scopus.com/inward/record.url?scp=85091567699&partnerID=8YFLogxK
U2 - https://doi.org/10.1056/NEJMoa1915035
DO - https://doi.org/10.1056/NEJMoa1915035
M3 - Article
C2 - 32877578
SN - 0028-4793
VL - 383
SP - 1242
EP - 1247
JO - New England journal of medicine
JF - New England journal of medicine
IS - 13
ER -