囊性纤维化治疗的全球化。

IF 2.2 3区 医学 Q2 PEDIATRICS
Current opinion in pediatrics Pub Date : 2025-06-01 Epub Date: 2025-03-27 DOI:10.1097/MOP.0000000000001458
Carlos E Milla
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引用次数: 0

摘要

综述目的:囊性纤维化领域正经历着巨大的变化,从CFTR基因克隆之日起积累的大量科学知识的转化已经导致了纠正基本缺陷的有效治疗方法的确定。这也使低收入和中等收入国家(LMICs)的护理提供者和囊性纤维化患者对囊性纤维化护理更加了解和熟练。近期发现:本综述主要关注与了解囊性纤维化在中低收入国家的现状高度相关的两个主要方面:对囊性纤维化普遍发生的认识,以及世界不同地区的不同发病率,以及国际合作努力传播最佳护理实践,试图缩小护理差距。摘要:随着这一领域继续迅速变化,多项国际努力正试图缩小富裕国家与中低收入国家之间明显存在的差距和差距。然而,这些努力受到有效治疗方法的限制,最严重的是CFTR调节药物的限制。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The globalization of cystic fibrosis care.

Purpose of review: The field of cystic fibrosis is experiencing dramatic changes, as the translation of a massive body of scientific knowledge accumulated from the day of the cloning of the CFTR gene has led to the identification of effective therapies to correct the basic defect. This has also allowed care providers and people with cystic fibrosis in low-income and middle-income countries (LMICs) to become more knowledgeable and proficient in cystic fibrosis cares.

Recent findings: This review focuses on two main aspects highly relevant to understand the current status of cystic fibrosis in LMICs: The recognition of the universal occurrence of cystic fibrosis, as well as the varying incidence in different regions of the world, and the collaborative international efforts for dissemination of best care practices as an attempt to close gaps in care.

Summary: As the field continues to change rapidly, multiple international efforts are attempting to close gaps and disparities clearly apparent between affluent countries and LMICs. However, these efforts are seriously hampered by limited access to effective therapies and most dramatically to CFTR modulator drugs.

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来源期刊
CiteScore
6.20
自引率
0.00%
发文量
184
审稿时长
6-12 weeks
期刊介绍: ​​​​​Current Opinion in Pediatrics is a reader-friendly resource which allows the reader to keep up-to-date with the most important advances in the pediatric field. Each issue of Current Opinion in Pediatrics contains three main sections delivering a diverse and comprehensive cover of all key issues related to pediatrics; including genetics, therapeutics and toxicology, adolescent medicine, neonatology and perinatology, and orthopedics. Unique to Current Opinion in Pediatrics is the office pediatrics section which appears in every issue and covers popular topics such as fever, immunization and ADHD. Current Opinion in Pediatrics is an indispensable journal for the busy clinician, researcher or student.
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