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本文引用的文献

1
Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.依伐卡托与泰比卡托和艾克卡托三联复方药物治疗携带单个 F508del 突变的囊性纤维化
N Engl J Med. 2019 Nov 7;381(19):1809-1819. doi: 10.1056/NEJMoa1908639. Epub 2019 Oct 31.
2
Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study.依伐卡托特治疗 12 至<24 月龄伴有 CFTR 门控突变(ARRIVAL)的囊性纤维化患儿:一项 3 期单臂研究。
Lancet Respir Med. 2018 Jul;6(7):545-553. doi: 10.1016/S2213-2600(18)30202-9. Epub 2018 Jun 7.
3
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study.评估长期使用组合 Lumacaftor 和 Ivacaftor 疗法治疗囊性纤维化纯合子 F508del-CFTR 突变患者的安全性和疗效(PROGRESS):一项 3 期扩展研究。
Lancet Respir Med. 2017 Feb;5(2):107-118. doi: 10.1016/S2213-2600(16)30427-1. Epub 2016 Dec 21.
4
Sustained Benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data.临床试验和囊性纤维化患者注册数据相结合证明伊伐卡托持续获益。
Am J Respir Crit Care Med. 2015 Oct 1;192(7):836-42. doi: 10.1164/rccm.201503-0578OC.

CFTR 调节剂:囊性纤维化的变革性治疗方法。

CFTR modulators: transformative therapies for cystic fibrosis.

机构信息

Cystic Fibrosis Foundation, Bethesda, MD.

出版信息

J Manag Care Spec Pharm. 2021 Feb;27(2):281-284. doi: 10.18553/jmcp.2021.27.2.281.

DOI:10.18553/jmcp.2021.27.2.281
PMID:33506726
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10391283/
Abstract

No funding contributed to the writing of this commentary. Both authors are employed by the Cystic Fibrosis Foundation. The Cystic Fibrosis Foundation has entered into therapeutic development award agreements and licensing agreements to assist with the development of CFTR modulators that may result in intellectual property rights, royalties, and other forms of consideration provided to CFF. Some of these agreements are subject to confidentiality restrictions and, thus, CFF cannot comment on them.

摘要

本评论文章的撰写未获得任何资金支持。两位作者均受雇于囊性纤维化基金会。囊性纤维化基金会已签订治疗开发奖励协议和许可协议,以协助开发 CFTR 调节剂,这可能导致向囊性纤维化基金会提供知识产权、版税和其他形式的对价。其中一些协议受保密限制,因此囊性纤维化基金会无法对此发表评论。