On-demand treatment with the experimental oral medication sebetralstat was effective for resolving swelling attacks in people with hereditary angioedema (HAE) — meeting all endpoints, or goals, in the Phase 3 KONFIDENT trial — top-line data show. Based on the positive findings, sebetralstat’s developer KalVista Pharmaceuticals is planning…
A 60-year-old man with hereditary angioedema (HAE) caused by an unknown mutation developed visual loss due to swelling of the optic nerve in both eyes, according to a case reported in Italy. His symptoms eased after starting treatment with Berinert (human C1 esterase inhibitor), an intravenous (into-the-vein) C1…
Three genetic regions were found to be associated with the risk of developing angioedema as a side effect of angiotensin-converting enzyme (ACE) inhibitors, according to a genome-wide association study (GWAS) of European and African-American people. These include a new genetic locus that hadn’t been…
Intellia Therapeutics’ experimental gene-editing therapy NTLA-2002 reduced the number of monthly swelling attacks by 95% among 10 people with hereditary angioedema (HAE) treated in the Phase 1 portion of a Phase 1/2 clinical trial, according to newly published data. The single-dose treatment also led to sustained, dose-dependent reductions in…
A 2-year-old girl with Down syndrome who was diagnosed with a rare form of hereditary angioedema (HAE) was the subject of a case report by researchers in Palestine. The child’s case was detailed in “Hereditary Angioedema With a Normal Complement…
The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on deucrictibant as a prophylactic, or preventive, treatment for hereditary angioedema (HAE) attacks. Deucrictibant developer Pharvaris said it will resume clinical testing of the therapy in HAE, including restarting the open-label extension part of the…
The investigational therapy donidalorsen was well tolerated and significantly reduced the rate of swelling attacks in hereditary angioedema (HAE) patients, meeting the main goal of the Phase 3 OASIS-HAE trial. Other secondary trial goals were also met, according to Ionis Pharmaceuticals, which is developing the treatment. The company…
Long-term preventive treatment with Haegarda reduced the number and severity of attacks and the use of rescue medications in people with hereditary angioedema (HAE), while improving their quality of life, a recent study has found. “These real-world findings indicate that long-term prophylaxis with [Haegarda] markedly improves important factors that…
In a 39-year-old man in France, hereditary angioedema (HAE) manifested with stroke-like symptoms, including weakness on one side of the body and difficulty speaking, despite treatment with danazol, a medication normally used to prevent HAE attacks. His symptoms eventually resolved with Takhzyro (lanadelumab), an approved therapy for…
Long-term use of attenuated androgens — male hormones used as a preventive treatment — may increase the risk for other disorders in hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE), a retrospective analysis suggests. Disorders linked with their use included high blood pressure, high cholesterol, diabetes, and benign liver tumors. “The results…
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