Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema.
case_report · Level V
Where this comes from
- Record sourced from PubMed, PMID 32877578.
- Also identified by DOI 10.1056/NEJMoa1915035.
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Abstract
Hereditary angioedema is characterized by recurrent and unpredictable episodes of subcutaneous and mucosal swelling that can be life threatening. IONIS-PKK-L<sub>Rx</sub> 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-PKK<sub>Rx</sub>, for 12 to 16 weeks, after which they received IONIS-PKK-L<sub>Rx</sub> 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.).
Medical subject headings
- Angioedemas, Hereditary
- Oligonucleotides, Antisense
- Prekallikrein