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How many billions is enough? Prioritizing profits over patients with cystic fibrosis.

作者信息

McGarry Meghan E, Gibb Elizabeth R, Laguna Theresa A, O'Sullivan Brian P, Sawicki Gregory S, Zobell Jeffery T

机构信息

Department of Pediatrics, Division of Pediatric Pulmonary Medicine, University of California San Francisco, San Francisco, California, USA.

Department of Pediatrics, Division of Pulmonary Medicine, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.

出版信息

Pediatr Pulmonol. 2023 May;58(5):1595-1597. doi: 10.1002/ppul.26335. Epub 2023 Feb 10.

DOI:10.1002/ppul.26335
PMID:36722731
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10121825/
Abstract
摘要

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How many billions is enough? Prioritizing profits over patients with cystic fibrosis.多少亿才算够?将利润置于囊性纤维化患者之上。
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[Challenges of personalized medicine for cystic fibrosis].[囊性纤维化个性化医疗面临的挑战]
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A new era of personalized medicine for cystic fibrosis - at last!囊性纤维化个性化医疗的新时代——终于到来了!
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引用本文的文献

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Affordable Cystic Fibrosis (CF) Transmembrane Conductance Regulator Modulator Drugs for CF: All CF Lives Worldwide Matter!用于囊性纤维化(CF)的价格亲民的囊性纤维化跨膜传导调节因子调节剂药物:全球所有囊性纤维化患者的生命都至关重要!
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Factors associated with frequent high-cost individuals with cystic fibrosis and their healthcare utilization and cost patterns.与囊性纤维化频繁高额费用个体及其医疗利用和费用模式相关的因素。
Sci Rep. 2023 Jun 1;13(1):8910. doi: 10.1038/s41598-023-35942-7.
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Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials.囊性纤维化患者接受 elexacaftor/tezacaftor/ivacaftor(ETI)治疗愿意参加随机调节剂和吸入性抗菌药物临床试验的意愿。
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Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis.依列卡福妥-替扎卡福妥-依伐卡托:用于囊性纤维化的改变人生的三联组合CFTR调节剂药物。
Pharmaceuticals (Basel). 2023 Mar 8;16(3):410. doi: 10.3390/ph16030410.

本文引用的文献

1
Current prices versus minimum costs of production for CFTR modulators.当前价格与 CFTR 调节剂的最低生产成本。
J Cyst Fibros. 2022 Sep;21(5):866-872. doi: 10.1016/j.jcf.2022.04.007. Epub 2022 Apr 16.
2
Left behind: The potential impact of CFTR modulators on racial and ethnic disparities in cystic fibrosis.遗留问题:CFTR 调节剂对囊性纤维化中种族和民族差异的潜在影响。
Paediatr Respir Rev. 2022 Jun;42:35-42. doi: 10.1016/j.prrv.2021.12.001. Epub 2021 Dec 22.
3
Rapid Improvement after Starting Elexacaftor-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and Advanced Pulmonary Disease.在患有囊性纤维化和晚期肺部疾病的患者中开始使用 Elexacaftor-Tezacaftor-Ivacaftor 后的快速改善。
Am J Respir Crit Care Med. 2021 Jul 1;204(1):64-73. doi: 10.1164/rccm.202011-4153OC.
4
The effectiveness and value of novel treatments for cystic fibrosis.新型囊性纤维化治疗方法的效果和价值。
J Manag Care Spec Pharm. 2021 Feb;27(2):276-280. doi: 10.18553/jmcp.2021.27.2.276.
5
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.
6
Pregnancy among cystic fibrosis women in the era of CFTR modulators.CFTR 调节剂时代囊性纤维化女性的妊娠问题。
J Cyst Fibros. 2017 Nov;16(6):687-694. doi: 10.1016/j.jcf.2017.01.008. Epub 2017 Feb 10.