New drugs in cystic fibrosis: what has changed in the last decade?

IF 4.3 3区 材料科学 Q1 ENGINEERING, ELECTRICAL & ELECTRONIC ACS Applied Electronic Materials Pub Date : 2022-05-21 eCollection Date: 2022-01-01 DOI:10.1177/20406223221098136
Juliana Roda, Catarina Pinto-Silva, Iris A I Silva, Carla Maia, Susana Almeida, Ricardo Ferreira, Guiomar Oliveira
{"title":"New drugs in cystic fibrosis: what has changed in the last decade?","authors":"Juliana Roda, Catarina Pinto-Silva, Iris A I Silva, Carla Maia, Susana Almeida, Ricardo Ferreira, Guiomar Oliveira","doi":"10.1177/20406223221098136","DOIUrl":null,"url":null,"abstract":"<p><p>Cystic fibrosis (CF), a life-limiting chronic disease caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene, affects more than 90,000 people worldwide. Until recently, the only available treatments were directed to symptom control, but they failed to change the course of the disease. New drugs developed in the last decade have the potential to change the expression, function, and stability of CFTR protein, targeting the basic molecular defect. The authors seek to provide an update on the new drugs, with a special focus on the most promising clinical trials that have been carried out to date. These newly approved drugs that target specific <i>CFTR</i> mutations are mainly divided into two main groups of CFTR modulators: potentiators and correctors. New therapies have opened the door for potentially disease-modifying, personalized treatments for patients with CF.</p>","PeriodicalId":3,"journal":{"name":"ACS Applied Electronic Materials","volume":null,"pages":null},"PeriodicalIF":4.3000,"publicationDate":"2022-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9128052/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"ACS Applied Electronic Materials","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/20406223221098136","RegionNum":3,"RegionCategory":"材料科学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2022/1/1 0:00:00","PubModel":"eCollection","JCR":"Q1","JCRName":"ENGINEERING, ELECTRICAL & ELECTRONIC","Score":null,"Total":0}
引用次数: 0

Abstract

Cystic fibrosis (CF), a life-limiting chronic disease caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene, affects more than 90,000 people worldwide. Until recently, the only available treatments were directed to symptom control, but they failed to change the course of the disease. New drugs developed in the last decade have the potential to change the expression, function, and stability of CFTR protein, targeting the basic molecular defect. The authors seek to provide an update on the new drugs, with a special focus on the most promising clinical trials that have been carried out to date. These newly approved drugs that target specific CFTR mutations are mainly divided into two main groups of CFTR modulators: potentiators and correctors. New therapies have opened the door for potentially disease-modifying, personalized treatments for patients with CF.

查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
治疗囊性纤维化的新药:在过去十年里发生了什么变化?
囊性纤维化(CF)是一种由囊性纤维化跨膜调节因子(CFTR)基因突变引起的限制生命的慢性疾病,影响全球超过9万人。直到最近,唯一可用的治疗方法都是针对症状控制,但它们未能改变疾病的进程。近十年来开发的新药有可能改变CFTR蛋白的表达、功能和稳定性,针对基本的分子缺陷。作者试图提供新药的最新情况,特别关注迄今为止进行的最有希望的临床试验。这些新批准的靶向特定CFTR突变的药物主要分为两大类CFTR调节剂:增强剂和校正剂。新的治疗方法为CF患者的潜在疾病改善和个性化治疗打开了大门。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 去求助
来源期刊
CiteScore
7.20
自引率
4.30%
发文量
567
期刊最新文献
Hyperbaric oxygen treatment promotes tendon-bone interface healing in a rabbit model of rotator cuff tears. Oxygen-ozone therapy for myocardial ischemic stroke and cardiovascular disorders. Comparative study on the anti-inflammatory and protective effects of different oxygen therapy regimens on lipopolysaccharide-induced acute lung injury in mice. Heme oxygenase/carbon monoxide system and development of the heart. Hyperbaric oxygen for moderate-to-severe traumatic brain injury: outcomes 5-8 years after injury.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1