Antisense inhibition of prekallikrein to control hereditary angioedema
New England Journal of Medicine Sep 29, 2020
Cohn DM, Viney NJ, Fijen LM, et al. - Researchers undertook a compassionate-use pilot study with two patients with severe bradykinin-mediated angioedema in order to determine the outcomes of treatment with IONIS-PKK-LRx, a ligand-conjugated antisense oligonucleotide designed for receptor-mediated delivery to hepatocytes, in these cases. These patients were initially given weekly subcutaneous injections of the unconjugated parent drug, IONIS-PKKRx, for 12 to 16 weeks, thereafter, patients were treated with IONIS-PKK-LRx at a dose of 80 mg every 3 to 4 weeks for 7 to 8 months. According to findings, a reduction in the angioedema attack rate was observed in association with the treatment.
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