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Treatment effects of Elexacaftor/Tezacaftor/Ivacaftor in people with CF carrying non-F508del mutations.

Author: Cohen-CymberknohMalena, DaganAdi, GurMichal, HechingMoshe, LivnatGalit, Mei-ZahavMeir, PraisDario, ShmueliEinat, ShteinbergMichal, SteinNili, Yaacoby-BianuKarin

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Overview

This study investigates the effectiveness of Elexacaftor/Tezacaftor/Ivacaftor (ETI) in treating cystic fibrosis (CF) patients with mutations other than F508del. Previous studies have shown ETI's potential to restore CFTR function in vitro, but its clinical efficacy in vivo has not been evaluated for non-F508del mutations. This study reports the results of ETI treatment in CF patients with non-F508del mutations.
Paper Details　
Original Abstract of the Article :
In vitro studies have demonstrated rescue of CFTR function with Elexacaftor/Tezacaftor/Ivacaftor (ETI) in several mutations other than F508del. However, clinical efficacy was not tested in vivo in people with CF (pwCF) carrying mutations other than F508del. We report effects of treatment with ETI in...See full text at original site
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引用元：
https://doi.org/10.1016/j.jcf.2022.10.011

データ提供：米国国立医学図書館（NLM）

Expanding the Reach of CFTR Modulators

Cystic fibrosis (CF), a genetic disorder affecting the lungs and other organs, continues to pose a significant challenge to healthcare professionals. This study investigates the effects of Elexacaftor/Tezacaftor/Ivacaftor (ETI), a combination therapy known as a CFTR modulator, in individuals with CF carrying non-F508del mutations. While previous studies had demonstrated the efficacy of ETI in individuals with F508del mutations, its impact on other mutations remained largely unexplored.

ETI: A Potential Solution for a Wider Range of CF Mutations

This study, although preliminary, suggests that ETI could potentially benefit individuals with CF carrying non-F508del mutations. This finding expands the potential therapeutic application of CFTR modulators, offering hope for a broader range of CF patients.

Navigating the Landscape of CF Treatment

This study highlights the ongoing research efforts to develop more effective treatments for CF. Understanding the complexities of this genetic disorder and identifying therapies that target different mutations is crucial for improving the lives of individuals living with CF.

Dr. Camel's Conclusion

This study expands the horizon of CF treatment, like a desert explorer discovering new oases. ETI's potential to benefit individuals with non-F508del mutations offers a promising avenue for improving the lives of a wider range of CF patients. This research underscores the importance of continuous innovation in medical research and the relentless pursuit of treatments for debilitating genetic disorders.

Date :
  1. Date Completed 2023-06-12
  2. Date Revised 2023-06-12
Further Info :

Pubmed ID

36372699

DOI: Digital Object Identifier

10.1016/j.jcf.2022.10.011

SNS
PICO Info
in preparation
Languages

English

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