Results 61 to 70 of about 6,532 (197)

Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review

, 2021
Cystic fibrosis (CF) is a rare, progressive, multi-organ genetic disease. Ivacaftor, a small-molecule CF transmembrane conductance regulator modulator, was the first medication to treat the underlying cause of CF.
Keval Chandarana, Fosca De Iorio, Jamie Duckers, Eleanor Lucas, Teja Thorat, Beth Lesher, Lisa J. McGarry   +6 more
core   +1 more source

Physiologically‐based pharmacokinetic modeling of the drug–drug interaction between ivacaftor and lefamulin in cystic fibrosis patients

CPT: Pharmacometrics & Systems Pharmacology
Lefamulin is being evaluated as a treatment for bacterial exacerbations in cystic fibrosis (CF). Ivacaftor is approved for the treatment of patients with CF.
Magali Amiel, Alice Ke, Steven P. Gelone, Hannah M. Jones, Wolfgang Wicha   +4 more
doaj   +1 more source

The effect of CFTR modulators on a cystic fibrosis patient presenting with recurrent pancreatitis in the absence of respiratory symptoms: a case report

BMC Gastroenterology, 2019
Background Cystic fibrosis (CF) is a genetic disorder of the epithelial CFTR apical chloride channel resulting in multi-organ manifestations, including pancreatic exocrine secretion.
J. Dixon Johns, Steven M. Rowe
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

Efectividad de ivacaftor en vida real en niños con fibrosis quística y mutación G551D

Anales de Pediatría, 2019
Resumen: Introducción: Ivacaftor es un potenciador de la proteína reguladora de la conductancia transmembrana de la fibrosis quística (CFTR) que ha demostrado en ensayos clínicos mejoría del estado nutricional y la función pulmonar de pacientes con ...
David Gomez-Pastrana, Chinedu Nwokoro, Mike McLean, Sarah Brown, Nanna Christiansen, Caroline S. Pao   +5 more
doaj   +1 more source

Ivacaftor for cystic fibrosis

BMJ, 2018
### What you need to know A 33 year old man with cystic fibrosis has come to discuss a new therapy for which he is eligible because of his genotype Phe508del/Gly551Asp. He has moderate lung function impairment with a forced expiratory volume in one second (FEV1) of 67% of predicted, which has declined from 85% over the preceding five years.
Barry, Peter J., Donaldson, Anna L., Jones, Andrew M.   +2 more
openaire   +3 more sources

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, Volume 36, Issue 36, 4 May 2026.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Bioengineered 3D hPSC‐Cholangiocyte Ducts With Physiological Signals for Biliary Disease Modeling

Advanced Healthcare Materials, Volume 15, Issue 20, 27 May 2026.
Tian and colleagues generated a bioengineered bile duct from human pluripotent stem cell (hPSC)‐derived intrahepatic cholangiocytes within a high‐throughput, 384‐well platform to systematically examine the influence of biliary physiological signals including fluid flow, stromal cells and bile acids, and models intrahepatic biliary disease progression ...
Britney Tian, Mina Ogawa, Manami Kondo, Gabrielle Langeveld, Ling Jun Huan, Feng Zhang, Andrew Hollinger, Sara Deir, Christine Bear, Boyang Zhang, Shinichiro Ogawa   +10 more
wiley   +1 more source

Ivacaftor and sinonasal pathology in a cystic fibrosis patient with genotype deltaF508/S1215N

, 2015
In patients with Cystic Fibrosis and a type III mutation, ivacaftor (Kalydeco(®), Vertex) can increase the opening time of the CFTR channel and improve chloride transport.
Sprij, A. J., Maaike Berkhout, Fokkens, W. J., Wytske Fokkens, Harry Heijerman, Heijerman, H. G. M., Charlotte Vreede, Vreede, C. L., Berkhout, M. C., Arwen Sprij   +9 more
core   +1 more source

Repurposing ivacaftor to attenuate LPS-induced acute lung injury: evidence from a non-cystic fibrosis model

Frontiers in Pharmacology
In this study, we explicitly evaluate the anti-inflammatory effects of ivacaftor as a route-of-delivery comparison (intraperitoneal vs. intratracheal) in a reproducible LPS-induced lung inflammation model. We evaluated the effects of ivacaftor [40 mg/kg,
Xiaoxuan Han, Yimin Zhu, Christopher W. Armstrong, Danni Li, Rachel McQuade, Andrew Jarnicki, Elena K. Schneider-Futschik   +6 more
doaj   +1 more source