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Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on CFTR Function in Patients with Cystic Fibrosis and One or Two <i>F508del</i> Alleles.

Author: BergesJulian, DittrichAnna-Maria, DrescherMarika, GraeberSimon Y, HirtzStephanie, KlajdaJan, MallMarcus A, MinsoRebecca, NaehrlichLutz, PallenbergSophia T, RingshausenFelix C, RohrbachAlexander, Rueckes-NilgesClaudia, ScheuermannHeike, SommerburgOlaf, StahlMirjam, TümmlerBurkhard, VitzthumConstanze, YuYin

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Observational StudyResearch that observes and collects data on participants' behavior and outcomes in their natural state, without intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA), a combination of CFTR modulators, significantly improved CFTR function in both airway and intestinal epithelia in patients with cystic fibrosis and one or two F508del alleles. The study demonstrated the effectiveness of ELX/TEZ/IVA in restoring CFTR function and its potential for improving clinical outcomes.
Paper Details　
Original Abstract of the Article :
<b>Rationale:</b> The CFTR (cystic fibrosis transmembrane conductance regulator) modulator combination elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to improve clinical outcomes and sweat chloride concentration in patients with cystic fibrosis (CF) and one or two <i>F508del</i> alleles. ...See full text at original site
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引用元：
https://doi.org/10.1164/rccm.202110-2249OC

データ提供：米国国立医学図書館（NLM）

Elexacaftor/Tezacaftor/Ivacaftor: A Beacon of Hope for Cystic Fibrosis

Cystic fibrosis (CF), a genetic disorder that affects the lungs, digestive system, and other organs, is a challenging condition to manage. Researchers are constantly searching for new treatments and therapies to improve the lives of those affected. This study investigates the effects of the CFTR modulator combination elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on CFTR function in patients with CF and one or two F508del alleles.

ELX/TEZ/IVA: A Promising Treatment for CF

The study demonstrates that ELX/TEZ/IVA effectively improves CFTR function in both airway and intestinal epithelia in patients with CF and one or two F508del alleles. The researchers observed significant improvements in CFTR-mediated chloride secretion, leading to a reduction in sweat chloride concentration and improvements in lung function.

Implications for CF Treatment

The findings suggest that ELX/TEZ/IVA holds significant promise as a treatment option for CF patients with F508del alleles. However, further research is needed to fully understand the long-term effects of this therapy and to assess its efficacy in patients with other CF mutations.

Dr.Camel's Conclusion

The quest for effective treatments for CF is an ongoing journey through the desert of scientific exploration. This study offers a glimmer of hope, showcasing the potential of ELX/TEZ/IVA to improve the lives of those living with this challenging condition. As we continue to explore new frontiers in CF research, we strive to create a brighter future for those affected by this disease.

Date :
  1. Date Completed 2022-04-22
  2. Date Revised 2022-04-22
Further Info :

Pubmed ID

34936849

DOI: Digital Object Identifier

10.1164/rccm.202110-2249OC

SNS
PICO Info
in preparation
Languages

English

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