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. In this phas...

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Veröffentlicht in:	The New England journal of medicine Jg. 391; H. 1; S. 21 - 31
Hauptverfasser:	Riedl, Marc A., Tachdjian, Raffi, Lumry, William R., Craig, Timothy, Karakaya, Gül, Gelincik, Asli, Stobiecki, Marcin, Jacobs, Joshua S., Gokmen, Nihal M., Reshef, Avner, Gompels, Mark M., Manning, Michael E., Bordone, Laura, Newman, Kenneth B., Treadwell, Sabrina, Wang, Sophie, Yarlas, Aaron, Cohn, Danny M.
Format:	Journal Article
Sprache:	Englisch
Veröffentlicht:	United States Massachusetts Medical Society 04.07.2024
Schlagworte:	Adolescent Adult Adverse events Aged Allergy Angioedema Angioedemas, Hereditary - drug therapy Antisense oligonucleotides Antisense therapy Child Clinical Medicine Clinical Medicine General Double-Blind Method Emergency medical care Emergency Medicine Emergency Medicine General Erythema Female Generalized linear models Genetics Genetics General Hospital-Based Clinical Medicine Humans Immunology Immunology General Inflammatory Disease Injections, Subcutaneous Male Middle Aged Missing data Oligonucleotides, Antisense - adverse effects Oligonucleotides, Antisense - therapeutic use Outpatient-Based Clinical Medicine Patients Pediatrics Pediatrics General Placebos Prophylaxis Quality of Life Rheumatology Rheumatology General Rhinopharyngitis Vasculitis Young Adult
ISSN:	0028-4793, 1533-4406, 1533-4406
Online-Zugang:	Volltext
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