Results 61 to 70 of about 3,564 (173)

Impact of elexacaftor-tezacaftor-ivacaftor in lung transplantation for cystic fibrosis in the United States

JHLT Open
Background: Cystic fibrosis (CF) is an autosomal recessive condition leading to progressive lung disease and often necessitating lung transplantation. Historically, CF has been one of the leading indications for lung transplants in the United States. The
Tahuanty A. Pena, MD, MS, Brittany Wright, PharmD, Kalpaj R. Parekh, MBBS, Julia Kleney-Tait, MD, PhD   +3 more
doaj   +1 more source

Cystic fibrosis-related diabetes in the era of modern treatment using CFTR modulators in pediatric patients—a systematic review

Frontiers in Pediatrics
BackgroundCystic fibrosis-related diabetes (CFRD) is a common comorbidity in cystic fibrosis (CF), particularly in the pediatric population. As cystic fibrosis transmembrane conductance regulator modulators (CFTRm) become widely used, there is growing ...
Anna Pietrzykowska, Jerzy Darmoń, Katarzyna Szymocha, Wiktoria Donocik, Mateusz Tarasiewicz, Julia Stadnik, Przemysława Jarosz-Chobot   +6 more
doaj   +1 more source

Comparison of Single‐Breath and Multi‐Breath Xe‐MRI in the Longitudinal Assessment of Treatment in Children With Cystic Fibrosis

Journal of Magnetic Resonance Imaging, Volume 63, Issue 6, Page 1652-1661, June 2026.
ABSTRACT Background Elexacaftor/tezacaftor/ivacaftor (ETI) is a current standard therapy for pediatric cystic fibrosis (CF). Multiple‐breath washout 129Xe MRI (MBW Xe‐MRI) is improved following 1 month of treatment. However, the utility of MBW Xe‐MRI over extended ETI treatment and its comparison to single‐breath Xe‐MRI and pulmonary function tests ...
Faiyza S. Alam, Samal Munidasa, Brandon Zanette, Daniel Li, Sharon Braganza, Felix Ratjen, Giles Santyr   +6 more
wiley   +1 more source

Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis [PDF]

, 2017
BACKGROUND Cystic fibrosis is an autosomal recessive disease caused by mutations in the CFTR gene that lead to progressive respiratory decline. Some mutant CFTR proteins show residual function and respond to the CFTR potentiator ivacaftor in vitro ...
Han, Linda, Ingenito, Edward P., Wilson, John, Ringshausen, Felix C., Kerem, Eitan, Ringshausen, Felix C, Ingenito, Edward P, Cori Daines, Felix C. Ringshausen, Edward P. Ingenito, Steven M. Rowe, John Wilson, Rowe, Steven M, Simard, Christopher, Elizabeth Tullis, Julie Lekstrom-Himes, Christopher Simard, Jane C. Davies, Charlotte McKee, Davies, Jane C, Mckee, Charlotte, Tullis, Elizabeth, Linda Han, Nair, Nitin, Eitan Kerem, Lekstrom-Himes, Julie, Davies, Jane C., Nitin Nair, Daines, Cori, Rowe, Steven M.   +29 more
core   +1 more source

Clinical Use of Home Spirometry in Children With Cystic Fibrosis

Pediatric Pulmonology, Volume 61, Issue 6, June 2026.
ABSTRACT Background The use of home spirometry (HSPIR) has increased in pediatric cystic fibrosis (CF) care, but how it has been used clinically and its impact on clinical care have not been described. The purpose of this study was to address this knowledge gap through a secondary analysis of data from a HSPIR quality improvement project to ...
Lucy Tan, Andrew Borowiec, James A. Reed, Mark Rath, Reid Masi, Laura Bennett, Don B. Sanders, Daniel J. Weiner, Clement L. Ren   +8 more
wiley   +1 more source

Longitudinal profiling of the intestinal microbiome in children with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor

mBio
The intestinal microbiome influences growth and disease progression in children with cystic fibrosis (CF). Elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA), the newest pharmaceutical modulator for CF, restores the function of the pathogenic mutated CF ...
Seth A. Reasoner, Rachel Bernard, Adam Waalkes, Kelsi Penewit, Janessa Lewis, Andrew G. Sokolow, Rebekah F. Brown, Kathryn M. Edwards, Stephen J. Salipante, Maria Hadjifrangiskou, Maribeth R. Nicholson   +10 more
doaj   +1 more source

Novel reaction to new cystic fibrosis medication Trikafta

Clinical Case Reports, 2021
We present a novel case of an urticaria multiforme‐type drug reaction to the new cystic fibrosis medication Trikafta (elexacaftor + tezacaftor + ivacaftor).
Julian Stashower, Patrick Carr, Virginia Miller, Barrett Zlotoff   +3 more
doaj   +1 more source

Performance Evaluation of a Novel Cystic Fibrosis Caregiver Burden Measure Demonstrates Significant Challenges for Parents of Children With CF During the Early Years: The Irish Comparative Outcomes Study (ICOS)

Pediatric Pulmonology, Volume 61, Issue 6, June 2026.
ABSTRACT Background As a part of the Irish Comparative Outcome national cohort study of childhood CF, this cross‐sectional study investigated challenges faced by parents of children with CF (CWCF) in the Republic of Ireland using a newly validated modified tool. Methods Parents completed the modified “Challenge of Living with Cystic Fibrosis‐Short Form”
Rini Bhatnagar, Nancy Bhardwaj, Gwinyai Masukume, Ricardo Segurado, Barry Linnane, Mary Herzig, Muireann Ni Chronin, David Mullane, Des W. Cox, Paul McNally, Sheila Javadpour, Ruth O'Reilly, Basil Elnazir, Peter Greally, Fiona Healy, Michael Williamson, Dubhfeasa Slattery, Gareth McCray, Gillian Lancaster, Claire Glasscoe, Kevin W. Southern, Laura Kirwan, Patricia Fitzpatrick   +22 more
wiley   +1 more source

Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles

ERJ Open Research, 2019
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene (CFTR) that result in diminished quantity and/or function of the CFTR anion channel.
Jennifer L. Taylor-Cousar, Marcus A. Mall, Bonnie W. Ramsey, Edward F. McKone, Elizabeth Tullis, Gautham Marigowda, Charlotte M. McKee, David Waltz, Samuel M. Moskowitz, Jessica Savage, Fengjuan Xuan, Steven M. Rowe   +11 more
doaj   +1 more source

Real-world population pharmacokinetics of tezacaftor-ivacaftor in children with cystic fibrosis:The SYM-CF study [PDF]

Aims: The clinical effectiveness of tezacaftor-ivacaftor in children with cystic fibrosis (cwCF) varies; some patients respond while others do not or have adverse effects.
Vonk, Steffie E.M., Mathôt, Ron A.A., Terheggen-Lagro, Suzanne W.J., Maitland–van der Zee, Anke Hilse, Haarman, Eric G., Janssens, Hettie M., Kemper, E. Marleen   +6 more
core   +1 more source