A promising oral treatment for hereditary angioedema with normal C1-inhibitor

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BioCryst Pharmaceuticals presented new evidence at the European Academy of Allergy and Clinical Immunology Congress in Valencia, Spain, showing that oral berotralstat reduced monthly attack rates in patients with hereditary angioedema (HAE-nC1-INH) who have normal C1-inhibitor levels. A case series of six patients showed a 75 to 100 percent reduction in attack rates after six months of treatment with berotralstat, with minimal adverse effects. Additionally, a study presented adverse health outcomes associated with prophylactic use of attenuated androgens in HAE, emphasizing the need for safer and more tolerable treatment options. The results support guidelines recommending targeted therapies for first-line long-term prophylaxis in HAE.

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