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donidalorsen
Indications:
- prophylactic treatment for hereditary angioedema
Dosage:
- 80 mg subcutaneously every 4 weeks
Adverse effects:
- erythema at the injection site
- headache
- nasopharyngitis
* 98% of adverse events were mild or moderate in severity.
Mechanism of action:
- antisense oligonucleotide, specifically reduces prekallikrein expression
General
short interfering double-stranded RNA; antisense oligonucleotide (siRNA, microRNA, miRNA)
References
- Lou N
Hereditary Angioedema Attacks Reduced With Donidalorsen Prophylaxis.
Investigational treatment passes muster in phase III trial
MedPage Today May 31, 2024
https://www.medpagetoday.com/allergyimmunology/allergy/110412
- Riedl MA, Tachdjian R, Lumry WR et al
Efficacy and Safety of Donidalorsen for Hereditary Angioedema.
N Engl J Med 2024. May 31
PMID: 38819395
https://www.nejm.org/doi/full/10.1056/NEJMoa2402478