Effectiveness of lanadelumab in patients with hereditary angioedema with normal C1 inhibitor and FXII mutation

Abstract

Hereditary angioedema (HAE) is a rare, autosomal dominant disorder, manifesting as recurrent and unpredictable episodes (attacks) of subcutaneous or submucosal edema of the face, limbs, genitals, gastrointestinal tract or larynx, triggered by local overproduction of bradykinin. The abdominal attacks are associated with nausea, vomiting, and pain, whereas those of the larynx are life threatening and may lead to death by asphyxiation, if not promptly treated.1,2