The New England journal of medicine
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Randomized Controlled Trial Multicenter Study
Efficacy and Safety of Donidalorsen for Hereditary Angioedema.
Hereditary angioedema is a rare disorder characterized by episodic, potentially life-threatening swelling caused by kallikrein-kinin dysregulation. Long-term prophylaxis can stabilize this system. Donidalorsen, an antisense oligonucleotide, specifically reduces prekallikrein expression. ⋯ Donidalorsen treatment reduced the hereditary angioedema attack rate, a finding that supports potential prophylactic use for hereditary angioedema. (Funded by Ionis Pharmaceuticals; OASIS-HAE ClinicalTrials.gov number, NCT05139810.).
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Editorial Review
New Therapies for Type 1 and Type 2 Hereditary Angioedema.