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Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…

Treatment with sebetralstat, an investigational and oral on-demand therapy, resulted in rapid relief for both abdominal and peripheral attacks in adults with hereditary angioedema (HAE), according to a new analysis Phase 2 trial data. Sebetralstat (formerly KVD900), by KalVista Pharmaceuticals, is a small molecule inhibitor of the…

Switching from under-the-skin injections of Takhzyro (lanadelumab) to oral capsules of Orladeyo (berotralstat) was generally safe and kept the occurrence of swelling attacks at low rates over one year in people with hereditary angioedema (HAE), according to a new data analysis of the Phase 2/3 APeX-S trial.

Most young children with hereditary angioedema (HAE) in the open-label Phase 3 SPRING study were free of swelling attacks following a year of treatment with Takhzyro (lanadelumab). That’s according to new data recently shared at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2022 in…

Many people with hereditary angioedema (HAE) have non-swelling symptoms, such as rash or fatigue, even when they aren’t having an acute swelling attack, a new study highlights. “HAE symptoms are not limited to only those experienced during HAE swelling attacks. Many different symptoms (including abdominal symptoms, pain, headache, fatigue, depression, and…

An approved therapy for multiple sclerosis (MS), called Gilenya (fingolimod), was effective at treating a man who had a rare combination of MS and hereditary angioedema (HAE), researchers in Greece have found. “We report [a] case study of a patient with concurrence of HAE and MS, discussing…

More than one-third of people with hereditary angioedema (HAE) hesitate to have children of their own — often due to fears of passing on the chronic genetic disease — according to data from a small survey. “Our study showed that HAE has a clear impact on reproductive decision-making,” the…

Firazyr (icatibant injection) appears to be at least as safe and effective at controlling swelling attacks in hereditary angiodema (HAE) patients with normal C1 inhibitor (C1-INH) activity as among those with reduced or no C1-INH activity, according to real-world data from Brazilian patients. Most attacks were successfully treated with…

Note: This story was updated June 22, 2022, to correct that the patient’s symptoms of angioedema resolved a few days after she stopped taking enalapril to treat her heart disease. A woman developed symptoms of angioedema after taking enalapril, a medicine commonly used to lower blood pressure and…

Sebetralstat, KalVista Pharmaceuticals‘ investigational on-demand treatment for swelling attacks in people with hereditary angioedema (HAE), led to a rapid and sustained suppression of kallikrein in HAE patients, according to data from a Phase 2 trial. As previously reported, the use of sebetralstat also was associated with fast…