Efficacy and Safety of Donidalorsen for Hereditary Angioedema.

Publication Date: 2024 Jul 04


Full Text Sources

Atypon

Ovid Technologies, Inc.

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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 Cohn

Abstract

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