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在等待下一代CFTR增强剂和校正剂的同时改善复杂的医疗护理:当前的治疗药物研发进程

Improving complex medical care while awaiting next-generation CFTR potentiators and correctors: The current pipeline of therapeutics.

作者信息

Goralski Jennifer L, Davis Stephanie D

机构信息

Division of Pulmonary and Critical Care Medicine, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.

Division of Pediatric Pulmonology, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.

出版信息

Pediatr Pulmonol. 2015 Oct;50 Suppl 40:S66-73. doi: 10.1002/ppul.23253.

Abstract

While a major target in cystic fibrosis (CF) research in recent years has been the development of corrector and potentiator drugs targeting the cystic fibrosis transmembrane conductance regulator (CFTR) protein, these therapies have not yet proven robust enough to replace or eliminate other therapies that have demonstrated improved health outcomes and quality of life in patients with CF. Further, ivacaftor is only indicated for approximately 5% of the US CF population, although the FDA has recently approved lumacaftor/ivacaftor, a combination therapy intended for those homozygous for Phe508del, which should reach a much larger number of patients. This review appraises therapeutics currently available or being studied while we await the next generation of CFTR potentiators and correctors.

摘要

尽管近年来囊性纤维化(CF)研究的一个主要目标是开发针对囊性纤维化跨膜传导调节因子(CFTR)蛋白的校正剂和增效剂药物,但这些疗法尚未被证明足够强大,足以取代或消除其他已证明能改善CF患者健康结局和生活质量的疗法。此外,依伐卡托仅适用于约5%的美国CF患者,尽管美国食品药品监督管理局(FDA)最近批准了鲁马卡托/依伐卡托,这是一种针对苯丙氨酸508缺失纯合子患者的联合疗法,预计将惠及更多患者。在我们等待下一代CFTR增效剂和校正剂出现的同时,本综述评估了目前可用或正在研究的治疗方法。

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