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http://dx.doi.org/10.1183/20734735.0016-2020 | DOI Listing |
Curr Opin Pulm Med
September 2025
4D Molecular Therapeutics, Emeryville, California, USA.
Purpose Of Review: There is a critical need for new therapies addressing the high unmet needs of individuals with rare lung diseases. This review examines the challenges industry sponsors face in developing therapeutic products for rare lung diseases, using cystic fibrosis as an example.
Recent Findings: Since the development of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, the drug development landscape for cystic fibrosis has changed.
Curr Opin Pulm Med
September 2025
Departments of Medicine and Pediatrics, Medical University of South Carolina, Charleston, South Carolina, USA.
Purpose Of Review: The advent of CFTR modulators and the adoption of telemedicine during the COVID-19 pandemic have prompted reconsideration of cystic fibrosis (CF) care models. This review explores how care delivery may evolve in response to these changes.
Recent Findings: Emerging evidence highlights the heterogeneity in response to CFTR modulators, with some patients continuing to experience disease progression.
Pediatr Pulmonol
September 2025
Rehabilitation Science PhD Program, University of Alabama at Birmingham, Birmingham, Alabama, USA.
Background: Exercise is an important part of a healthy lifestyle for people with cystic fibrosis (CF). Exercise testing is also recommended as part of a routine assessment to provide physical activity counseling/exercise prescription. Body composition assessment is also an important assessment that can also guide dietary counseling.
View Article and Find Full Text PDFNutrients
July 2025
Division of Gastroenterology, Hepatology & Nutrition, Department of Pediatrics, Nationwide Children's Hospital, The Ohio State University, Columbus, OH 43205, USA.
There is a well-established association between cystic fibrosis (CF) and malnutrition. Several comorbid conditions have also been associated with undernutrition in people with cystic fibrosis (PwCF). Highly effective modulator therapy has allowed for a paradigm shift altering disease progression and management.
View Article and Find Full Text PDFAnn Am Thorac Soc
August 2025
Cincinnati Children's Hospital Medical Center, Pediatrics & Radiology, Cincinnati, Ohio, United States;
Rationale: Elexacaftor/tezacaftor/ivacaftor (ETI) has significantly improved lung function in people with cystic fibrosis (CF), prompting the need for outcome measures that can detect mild disease. In this new era of CFTR modulator therapy, more sensitive endpoints are required to evaluate the progression of early lung disease and to determine the efficacy of new CF therapies. Prior to the availability of highly effective therapies 129Xenon magnetic resonance imaging (Xe MRI) was shown to be more sensitive to regional ventilation changes compared to spirometry.
View Article and Find Full Text PDF