Journal article
Understanding and addressing the needs of people with cystic fibrosis in the era of CFTR modulator therapy
The lancet respiratory medicine, Vol.11(10), pp.916-931
10/2023
DOI: 10.1016/S2213-2600(23)00324-7
PMID: 37699420
Abstract
Cystic fibrosis is a multiorgan disease caused by impaired function of the cystic fibrosis transmembrane conductance regulator (CFTR). Since the introduction of the CFTR modulator combination elexacaftor–tezacaftor–ivacaftor (ETI), which acts directly on mutant CFTR to enhance its activity, most people with cystic fibrosis (pwCF) have seen pronounced reductions in symptoms, and studies project marked increases in life expectancy for pwCF who are eligible for ETI. However, modulator therapy has not cured cystic fibrosis and the success of CFTR modulators has resulted in immediate questions about the new state of cystic fibrosis disease and clinical challenges in the care of pwCF. In this Series paper, we summarise key questions about cystic fibrosis disease in the era of modulator therapy, highlighting state-of-the-art research and clinical practices, knowledge gaps, new challenges faced by pwCF and the potential for future health-care challenges, and the pressing need for additional therapies to treat the underlying genetic or molecular causes of cystic fibrosis.
Details
- Title: Subtitle
- Understanding and addressing the needs of people with cystic fibrosis in the era of CFTR modulator therapy
- Creators
- Katherine B Hisert - National Jewish HealthSusan E Birket - University of Alabama at BirminghamJohn Paul Clancy - Cystic Fibrosis FoundationDamian G DowneyJohn F Engelhardt - University of IowaIsabelle Fajac - Université Paris CitéRobert D Gray - Centre for Inflammation ResearchMarrah E Lachowicz-Scroggins - National Heart Lung and Blood InstituteNicole Mayer-Hamblett - Seattle Children's HospitalPatrick Thibodeau - Cystic Fibrosis FoundationKatherine L Tuggle - Cystic Fibrosis FoundationClaire E Wainwright - The University of QueenslandKris De Boeck - KU Leuven
- Resource Type
- Journal article
- Publication Details
- The lancet respiratory medicine, Vol.11(10), pp.916-931
- DOI
- 10.1016/S2213-2600(23)00324-7
- PMID
- 37699420
- NLM abbreviation
- Lancet Respir Med
- ISSN
- 2213-2600
- eISSN
- 2213-2619
- Grant note
- DOI: 10.13039/100000060, name: National Institute of Allergy and Infectious Diseases; DOI: 10.13039/100000002, name: National Institutes of Health; DOI: 10.13039/100000062, name: National Institute of Diabetes and Digestive and Kidney Diseases; DOI: 10.13039/100000897, name: Cystic Fibrosis Foundation; DOI: 10.13039/100006108, name: NCATS; DOI: 10.13039/100000016, name: U.S. Department of Health and Human Services; DOI: 10.13039/100000050, name: National Heart, Lung, and Blood Institute
- Language
- English
- Electronic publication date
- 09/08/2023
- Date published
- 10/2023
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Anatomy and Cell Biology; Radiation Oncology; Internal Medicine
- Record Identifier
- 9984464475702771
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