Hereditary angioedema, a disabling condition, with high mortality when untreated, is caused by C1 inhibitor deficiency and other regulatory disorders of bradykinin production or metabolism. This review covers the remarkable progress made in biological therapies for this rare disorder.Areas covered: Over the past 10 years, several evidence-based parenteral treatments have been licensed, including two plasma-derived C1 inhibitor replacement therapies and one recombinant C1 inhibitor replacement for acute treatment of angioedema attacks and synthetic peptides for inhibition of kallikrein or bradykinin B2 receptors, with oral small molecule treatments currently in clinical trial. Moreover, recent advances in prophylaxis by subcutaneous C1 inhibitor to restore near-normal plasma function or by humanised antibody inhibition of kallikrein have resulted in freedom from symptoms for a high proportion of those treated.Expert opinion: This plethora of treatment possibilities has come about as a result of recent scientific advances. Collaboration between patient groups, basic and clinical scientists, physicians, nurses and the pharmaceutical industry has underpinned this translation of basic science into treatments and protocols. These in their turn have brought huge improvements in prognosis, quality of life and economic productivity to patients, their families and the societies in which they live.

中文翻译：

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遗传性血管性水肿的生物疗法：一种罕见疾病的转化。

遗传性血管性水肿是一种致残性疾病，未经治疗会导致很高的死亡率，这是由C1抑制剂缺乏和缓激肽产生或代谢的其他调节性疾病引起的。这项综述涵盖了针对这种罕见疾病的生物疗法所取得的显着进展。涉及的领域：过去10年中，已批准了几种基于证据的肠胃外治疗方法，包括两种血浆衍生的C1抑制剂替代疗法和一种重组C1抑制剂替代疗法。目前正在临床试验中的口服小分子疗法可用于对血管性水肿发作的急性治疗和抑制激肽释放酶或缓激肽B2受体的合成肽的治疗。此外，皮下C1抑制剂恢复近乎正常的血浆功能或人源化抗体抑制激肽释放酶的预防的最新进展已使大部分被治疗者摆脱了症状。专家意见：结果产生了如此众多的治疗可能性最近的科学进展。患者群体，基础和临床科学家，医师，护士和制药行业之间的合作为基础科学到治疗和方案的转化奠定了基础。这些又为患者，他们的家人和他们所生活的社会带来了预后，生活质量和经济生产力方面的巨大进步。由于最近的科学进步，出现了如此众多的治疗可能性。患者群体，基础和临床科学家，医师，护士和制药行业之间的合作为基础科学到治疗和方案的转化奠定了基础。这些又为患者，他们的家人和他们所生活的社会带来了预后，生活质量和经济生产力方面的巨大进步。由于最近的科学进步，出现了如此众多的治疗可能性。患者群体，基础和临床科学家，医师，护士和制药行业之间的合作为基础科学到治疗和方案的转化奠定了基础。这些又为患者，他们的家人和他们所生活的社会带来了预后，生活质量和经济生产力方面的巨大进步。