Efficacy and Safety of Donidalorsen for Hereditary Angioedema.
Publication Date: 2024 Jul 04
Full Text Sources
Authors
Marc A Riedl; Raffi Tachdjian; William R Lumry; Timothy Craig; Gül Karakaya; Asli Gelincik; Marcin Stobiecki; Joshua S Jacobs; Nihal M Gokmen; Avner Reshef; Mark M Gompels; Michael E Manning; Laura Bordone; Kenneth B Newman; Sabrina Treadwell; Sophie Wang; Aaron Yarlas; Danny M CohnAbstract
OBJECTIVE
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.
Source
The New England journal of medicine
Pub Types(s)
Journal Article
Language
English
PubMed ID
38819395