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Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis
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New and evolving therapies for hereditary angioedema

Sara Barmettler, Yu Li, Aleena Banerji

    Allergy and Asthma Proceedings
    |December 25, 2018
    PubMed

    Hereditary angioedema (HAE) is a rare, autosomal dominant, genetic disorder associated with a deficiency in C1 inhibitor protein. HAE is characterized by recurrent and unpredictable episodes of swelling of the extremities, abdomen, face, and upper airway. There are several newly approved drugs as well as investigational products that are currently under study for the management of patients with HAE, with the potential to optimize care and improve quality of life for patients with HAE. We reviewed the evolution of HAE treatment options in the United States and discussed mechanisms of action, routes of administration, and efficacy of these therapies.

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