Results 101 to 110 of about 6,532 (197)

Comparing the Effectiveness between Ivacaftor and GLPG 1837

, 2021
Cystic fibrosis is an autosomal disease that is caused by a defect in the CFTR, or cystic fibrosis transmembrane conductance regulator. It affects more than 70,000 people world-wide and is life-threatening to those affected.
Reyes, Saimi Vanessa, Sankavaram, Vibha, Carreras, Jordan Nichole   +2 more
core  

Lumacaftor-ivacaftor in the treatment of cystic fibrosis: design, development and place in therapy

Drug Design, Development and Therapy, 2019
GJ ConnettNational Institute for Health Research, Southampton Respiratory Biomedical Research Centre, University Hospital Southampton NHS Foundation Trust, Southampton, SO16 6YD, UKAbstract: Lumacaftor-ivacaftor is a combination of two small molecule ...
Connett GJ
doaj  

Impact of CFTR modulator concentrations on clinical response in cystic fibrosis. [PDF]

Eur Respir J
Chalamalla AR, Baker E, Ryan KJ, Dowell A, Natt JR, Zemanick ET, Konstan MW, Mayer-Hamblett N, Acosta EP, Guimbellot JS, CHEC-SC investigators.   +10 more
europepmc   +1 more source

Ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor–ivacaftor for treating cystic fibrosis: a systematic review and economic evaluation

Background Cystic fibrosis is a life-limiting genetic condition that affects over 9000 people in England. Cystic fibrosis is usually diagnosed through newborn screening and causes symptoms throughout the body, including the lungs and digestive system ...
Archie Walters, Benjamin G Farrar, Tracey Jhita, Victoria Wakefield, Isaac Mackenzie, Kate Ennis, Nicole Downes, Steven J Edwards   +7 more
core   +1 more source

Editorial: Real-world experience with CFTR modulator therapy

Frontiers in Pharmacology, 2023
Burkhard Tümmler, Burkhard Tümmler, Pierre-Régis Burgel, Pierre-Régis Burgel   +3 more
doaj   +1 more source

Altered functional interactions between CFTR disease mutants ΔF508 and G551D and the protein kinase A catalytic subunit. [PDF]

J Physiol
Závoti O, Simon MA, Csanády L.
europepmc   +1 more source

Urticaria multiforme-like eruption due to a novel agent elexacaftor/tezacaftor/ivacaftor in a pediatric patient with cystic fibrosis

JAAD Case Reports, 2021
Rebecca H. Goldberg, BS, Natalie H. Matthews, MD, MPhil, Alexandra C. Hristov, MD, Frank Wang, MD   +3 more
doaj   +1 more source

Off-label treatment with elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: real-world evidence from the German CF Registry. [PDF]

ERJ Open Res
Stahl M, Gerts FS, Hafkemeyer S, Wosniok J, Naehrlich L, German CF Registry of the Mukoviszidose e.V. and participating sites.   +5 more
europepmc   +1 more source

Predictive capacity of paediatric nasal epithelial cells in sequential CFTR modulator therapy. [PDF]

Thorax
Fawcett LK, Chew ZA, Schneider-Futschik EK, Allan KM, Patel HR, Jaffe A, Waters S.   +6 more
europepmc   +1 more source

Systematic Review of Nasal Endoscopy Scores in Cystic Fibrosis Patients Treated With Cystic Fibrosis Transmembrane Conductance Regulator Modulators. [PDF]

Pediatr Pulmonol
Gallardo R, Silva BS, Salgado LS, Ribeiro JD, Sakano E.   +4 more
europepmc   +1 more source