囊性纤维化的未来:全球视角。

IF 2.7 3区 医学 Q1 PEDIATRICS
Felix Ratjen
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引用次数: 0

摘要

囊性纤维化(CF)患者的肺部疾病和其他疾病表现的严重程度已得到显著改善,这些患者既有对使用 CF 跨膜调节器(CFTR)调节剂的小分子疗法有反应的突变,又有机会获得这些药物。遗憾的是,全球许多 CF 患者都无法获得这些药物,主要局限于高收入国家。对于那些有资格使用 CFTR 调节剂的患者来说,新的问题又出现了,那就是是否还需要其他药物来治疗 CF 肺部疾病,以及目前的护理模式是否需要严格监测。本文旨在总结CF护理在未来可能发生的变化,呼吁扩大高效药物的供应范围,让每一位CF患儿都能从治疗中受益。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The future of cystic fibrosis: A global perspective.

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.

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来源期刊
Pediatric Pulmonology
Pediatric Pulmonology 医学-呼吸系统
CiteScore
6.00
自引率
12.90%
发文量
468
审稿时长
3-8 weeks
期刊介绍: Pediatric Pulmonology (PPUL) is the foremost global journal studying the respiratory system in disease and in health as it develops from intrauterine life though adolescence to adulthood. Combining explicit and informative analysis of clinical as well as basic scientific research, PPUL provides a look at the many facets of respiratory system disorders in infants and children, ranging from pathological anatomy, developmental issues, and pathophysiology to infectious disease, asthma, cystic fibrosis, and airborne toxins. Focused attention is given to the reporting of diagnostic and therapeutic methods for neonates, preschool children, and adolescents, the enduring effects of childhood respiratory diseases, and newly described infectious diseases. PPUL concentrates on subject matters of crucial interest to specialists preparing for the Pediatric Subspecialty Examinations in the United States and other countries. With its attentive coverage and extensive clinical data, this journal is a principle source for pediatricians in practice and in training and a must have for all pediatric pulmonologists.
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