Abstract
Hereditary angioedema with normal C1-inhibitor (nC1-INH-HAE) is a very rare condition, characterized by recurrent and unpredictable swellings which are disabling and potentially fatal. There are currently no approved prophylactic treatments for this condition. In a phase 2 study, we assessed efficacy and safety following treatment with oligonucleotide antisense therapy targeted against plasma prekallikrein (PKK-LRx) in patients with nC1-INH-HAE.
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