Suppr超能文献

囊性纤维化的未来:全球视角。

The future of cystic fibrosis: A global perspective.

作者信息

Ratjen Felix

机构信息

Department of Pediatrics, Division of Respiratory Medicine, Ontario, Canada.

Translational Medicine Program, Research Institute, Hospital for Sick Children, Ontario, Canada.

出版信息

Pediatr Pulmonol. 2025 Mar;60 Suppl 1:S75-S76. doi: 10.1002/ppul.27337. Epub 2024 Oct 17.

Abstract

The severity of lung disease as well as other disease manifestations have dramatically improved in those patients with cystic fibrosis (CF) that both have mutations responsive to small molecule- based therapies with CF transmembrane regulator (CFTR) modulators and do have access to these drugs. Unfortunately, these medications are not available to many patients with CF across the globe with access largely limited to high income countries. For those eligible to CFTR modulators new questions have arisen regarding the ongoing need for other medications addressing CF lung disease as well current care models with tight monitoring. This article aims to summarize how CF care may change in the future making a plea to expand the availability of highly effective medications to every child with CF that could benefit from treatment.

摘要

对于那些患有囊性纤维化(CF)且具有对基于小分子疗法的CF跨膜调节因子(CFTR)调节剂有反应的突变并且能够获得这些药物的患者,肺部疾病以及其他疾病表现的严重程度已得到显著改善。不幸的是,全球许多CF患者无法获得这些药物,药物获取在很大程度上仅限于高收入国家。对于那些有资格使用CFTR调节剂的患者,出现了关于持续需要其他治疗CF肺部疾病的药物以及当前严格监测的护理模式的新问题。本文旨在总结CF护理在未来可能如何变化,呼吁扩大高效药物的可及性,使每个能从治疗中受益的CF患儿都能获得药物。

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验