Marisa Wexler, MS, senior science writer —

Japan’s Ministry of Health, Labor and Welfare (MHLW) has approved oral Orladeyo (berotralstat), at a daily dose of 150 mg, to prevent hereditary angioedema (HAE) attacks in individuals ages 12 and up. According to the therapy’s developer, BioCryst Pharmaceuticals, Orladeyo is the first prophylactic, or preventive, HAE medication approved…

Variations in the genes KNG1 and XPNPEP1 may be associated with hereditary angioedema (HAE) that occurs in people without any of the mutations that typically cause the disease, a new study suggests. The preliminary findings highlight the genetic complexity of HAE, researchers said. The study, “Deciphering…

Treatment with Takhzyro (lanadelumab) significantly improves health-related quality of life in people with hereditary angioedema (HAE), new data from its pivotal clinical trial show. While all dose regimens improved patient outcomes, the approved dose — 300 mg given every two weeks — provided the greatest benefits. Patients on this…

Assessing C1-inhibitor (C1-INH) activity for the diagnosis and monitoring of hereditary angioedema (HAE) can be done through three distinct validated methods with similar accuracy, a new study found. The study, “Parallel comparison of three methodologies for measuring functional C1-inhibitor in Hereditary angioedema patients,” was published in …

Preventive treatment with oral berotralstat (BCX7353) significantly lowers the frequency of swelling attacks in people with hereditary angioedema (HAE), newly published results from the APeX-2 clinical trial demonstrate. The study, “Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: a randomized, double-blind, placebo-controlled phase 3…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

The first documented case of a hereditary angioedema (HAE) attack in an unborn child — whose symptoms resolved after treating the mother with the recombinant human C1-inhibitor (rhC1-INH) therapy, Ruconest — has been reported. The case report, “Hereditary Angioedema Attack in Utero and Treatment of the Mother…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

Antisense oligonucleotides (ASOs) being developed by Ionis Pharmaceuticals may reduce the frequency of attacks in people with hereditary angioedema (HAE), a small study suggests. The study, “Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema,” funded by Amsterdam UMC, was published in The New England…

BioCryst Pharmaceuticals is prepared to launch Orladeyo (berotralstat), an oral treatment for hereditary angioedema (HAE), by the end of the year if the medication is approved, the company has announced. “We are currently in an exciting transformation from a company primarily focused on R&D [research and development]…