I am a
Home I AM A Search Login

Papers of the Week


Papers: 1 Jun 2024 - 7 Jun 2024


2024 May 31


N Engl J Med


38819395

Efficacy and Safety of Donidalorsen for Hereditary Angioedema.

Authors

Riedl MA, Tachdjian R, Lumry WR, Craig T, Karakaya G, Gelincik A, Stobiecki M, Jacobs JS, Gokmen NM, Reshef A, Gompels MM, Manning ME, Bordone L, Newman KB, Treadwell S, Wang S, Yarlas A, Cohn DM

Abstract

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.