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A systematic cochrane review of corrector therapies (with or without potentiators) for people with cystic fibrosis with class II gene variants (most commonly F508DEL).

作者信息

Southern K W, Murphy J, Sinha I P, Nevitt S J

机构信息

Department of Women's and Children's Health, University of Liverpool, Liverpool, UK.

Department of Health Data Science, University of Liverpool, Liverpool, UK.

出版信息

Paediatr Respir Rev. 2021 Jun;38:33-36. doi: 10.1016/j.prrv.2021.03.001. Epub 2021 Mar 11.

DOI:10.1016/j.prrv.2021.03.001
PMID:33875358
Abstract
摘要

相似文献

1
A systematic cochrane review of corrector therapies (with or without potentiators) for people with cystic fibrosis with class II gene variants (most commonly F508DEL).对患有II类基因变异（最常见的是F508DEL）的囊性纤维化患者使用校正疗法（有或没有增效剂）的Cochrane系统评价。
Paediatr Respir Rev. 2021 Jun;38:33-36. doi: 10.1016/j.prrv.2021.03.001. Epub 2021 Mar 11.
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Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis.囊性纤维化的增效剂（针对III类和IV类突变的特定疗法）。
Cochrane Database Syst Rev. 2015 Mar 26(3):CD009841. doi: 10.1002/14651858.CD009841.pub2.
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F508del-cystic fibrosis transmembrane regulator correctors for treatment of cystic fibrosis: a patent review.用于治疗囊性纤维化的F508del-囊性纤维化跨膜传导调节因子校正剂：专利综述
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Efficacy and Safety of CFTR Corrector and Potentiator Combination Therapy in Patients with Cystic Fibrosis for the F508del-CFTR Homozygous Mutation: A Systematic Review and Meta-analysis.CFTR 校正剂和增强剂联合治疗 F508del-CFTR 纯合突变囊性纤维化患者的疗效和安全性：系统评价和荟萃分析。
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Lumacaftor/ivacaftor, a novel agent for the treatment of cystic fibrosis patients who are homozygous for the F580del CFTR mutation.鲁马卡托/依伐卡托，一种用于治疗F580del囊性纤维化跨膜传导调节因子（CFTR）突变纯合子的囊性纤维化患者的新型药物。
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A systematic Cochrane Review of correctors (specific therapies for class II CFTR mutations) for cystic fibrosis.一项关于囊性纤维化校正剂（针对II类CFTR突变的特定疗法）的Cochrane系统评价。
Paediatr Respir Rev. 2019 Apr;30:25-26. doi: 10.1016/j.prrv.2019.01.003. Epub 2019 Feb 7.
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Potentiators for cystic fibrosis - targeting the underlying molecular defect.囊性纤维化的增效剂——针对潜在分子缺陷
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Corrector combination therapies for F508del-CFTR.F508del-CFTR 的校正组合疗法。
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Cystic fibrosis transmembrane regulator correctors and potentiators.囊性纤维化跨膜转导调节因子校正剂和增强剂。
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Cystic fibrosis transmembrane regulator potentiators as promising cystic fibrosis therapies.囊性纤维化跨膜转导调节因子增效剂作为有前途的囊性纤维化治疗方法。
Expert Opin Investig Drugs. 2011 Mar;20(3):423-5. doi: 10.1517/13543784.2011.554823. Epub 2011 Feb 9.

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Health Technol Assess. 2025 May;29(19):1-111. doi: 10.3310/CPLD8546.
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Factors associated with cystic fibrosis mortality before the age of 30: retrospective analysis of a cohort in southern Brazil.与 30 岁前囊性纤维化死亡相关的因素：巴西南部队列的回顾性分析。
Braz J Med Biol Res. 2024 Aug 23;57:e13476. doi: 10.1590/1414-431X2024e13476. eCollection 2024.
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CFTR Modulator Therapy and Its Impact on Lung Transplantation in Cystic Fibrosis.
囊性纤维化跨膜传导调节因子调节剂疗法及其对囊性纤维化肺移植的影响。
Pulm Ther. 2021 Dec;7(2):377-393. doi: 10.1007/s41030-021-00170-9. Epub 2021 Aug 18.