Results 11 to 20 of about 3,564 (173)

Physiologically Based Pharmacokinetic Modeling of Elexacaftor/Tezacaftor/Ivacaftor in Infants With Cystic Fibrosis [PDF]

CPT: Pharmacometrics & Systems Pharmacology
Ivacaftor is the only cystic fibrosis transmembrane conductance regulator modulator approved for infants ≥ 1 month. The elexacaftor/tezacaftor/ivacaftor combination, approved for children aged ≥ 2 years, has been shown to significantly slow CF ...
Ngoc Hoa Truong, Sihem Benaboud, Naïm Bouazza, Eric Deneuville, Anne Mornand, Philippe Reix, Mélanie Ribault, Pierre‐Régis Burgel, Léo Froelicher‐Bournaud, Steeve Rouillon, Mohammed Rohi Sanoufi, Gabrielle Lui, Saïk Urien, Jean‐Marc Tréluyer, Isabelle Sermet‐Gaudelus, Frantz Foissac, Modul‐CF study group   +16 more
doaj   +3 more sources

Pharmacokinetic and Drug–Drug Interaction Profiles of the Combination of Tezacaftor/Ivacaftor

Clinical and Translational Science, 2019
Drug–drug interaction (DDI) studies are described for tezacaftor/ivacaftor, a new cystic fibrosis transmembrane conductance regulator modulator therapy for the treatment of cystic fibrosis.
Varun Garg, Jinshan Shen, Chonghua Li, Sagar Agarwal, Asfiha Gebre, Sarah Robertson, Jiayin Huang, Linda Han, Licong Jiang, Kristin Stephan, Linda T. Wang, Julie Lekstrom‐Himes   +11 more
doaj   +2 more sources

Comparative efficacy and safety of CFTR modulators for people with cystic fibrosis with phe508del mutation: a systematic review and bayesian network meta-analysisResearch in context [PDF]

EClinicalMedicine
Summary: Background: The development of cystic fibrosis transmembrane conductance regulator (CFTR) modulators (correctors and potentiators) emerged as a promising approach, aiming to restore CFTR protein function. A lack of head-to-head trials comparing
Mohammed Safeer V S, Simran Behl, Pankaj C. Vaidya, Pawan Tiwari, Saroj Kundan Bharti, Najiya Nahan, Jitendra Kumar Sahu, Dipika Bansal   +7 more
doaj   +2 more sources

Exposure to CFTR Modulators During Pregnancy in Cystic Fibrosis: Four Cases to Highlight Neonatal Diagnostic Challenges and Outcomes [PDF]

International Journal of Neonatal Screening
CFTR modulators have transformed the clinical evolution of patients with CF. The number of pregnancies is increasing in women with CF, most of whom are now treated with CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ETI) or Tezacaftor ...
Louis Domenach, Adrien Pagin, Camille Cisterne, Marie-Pierre Audrezet, Laure Couderc, Laetitia Monteil, Léa Roditis, Marlène Murris, Julie Macey, Michael Fayon, Stéphanie Bui, Marie-Pierre Reboul   +11 more
doaj   +2 more sources

Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor

Molecules
The monogenic rare disease Cystic Fibrosis (CF) is caused by mutations in the gene encoding the CF transmembrane conductance (CFTR) protein, an anion channel expressed at the apical plasma membrane of epithelial cells.
Filipa C. Ferreira, Camilla D. Buarque, Miquéias Lopes-Pacheco   +2 more
doaj   +2 more sources

Large variations in total and allele-specific transcript expression in a disease mutation-independent manner [PDF]

Scientific Reports
Individuals with monogenic diseases, even those with identical disease-causing mutations, exhibit considerable clinical heterogeneity in severity and outcomes.
Moritz Freyberg, Merete Bewig, Giovana Bavia Bampi, Candela Manfredi, Disha Joshi, Robert Rauscher, Jeong S. Hong, Jörg Große-Onnebrink, Sivagurunathan Sutharsan, Florian Stehling, Ingrid Bobis, Manfred Ballmann, Eric J. Sorscher, Zoya Ignatova   +13 more
doaj   +2 more sources

Management of Infants Exposed to Prenatal and Postnatal Elexacaftor/Tezacaftor/Ivacaftor: A Cross-Sectional Survey. [PDF]

Health Sci Rep
Health Science Reports, Volume 9, Issue 5, May 2026.
Haskett H, Ren CL.
europepmc   +2 more sources

Improved Quality of Life in Children With Cystic Fibrosis Who Received Transmembrane Conductance Regulator Modulators. [PDF]

Acta Paediatr
ABSTRACT Aim Children with cystic fibrosis (CF) face substantial daily treatment burdens and the effects of transmembrane conductance regulator modulators on these have not been sufficiently described. We evaluated changes in treatment burden after elexacaftor tezacaftor ivacaftor (ETI) was initiated.
Svedberg M, Lundqvist F, Östrand HA, Krantz C, Ekman P, Imberg H.   +5 more
europepmc   +2 more sources

Real-life impact of highly effective CFTR modulator therapy in children with cystic fibrosis

Frontiers in Pharmacology, 2023
Introduction: Recently, cystic fibrosis transmembrane regulator modulator therapy with elexacaftor/tezacaftor/ivacaftor has become available for children with cystic fibrosis (CF) carrying at least one F508del mutation.Objective: To assess the ...
Margarete Olivier, Alexandra Kavvalou, Matthias Welsner, Raphael Hirtz, Svenja Straßburg, Sivagurunathan Sutharsan, Florian Stehling, Mathis Steindor   +7 more
doaj   +1 more source

Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials [PDF]

, 2023
Background Elexacaftor–tezacaftor–ivacaftor has been shown to be safe and efficacious in people with cystic fibrosis and at least one F508del allele. Our aim was to identify a novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator ...
Ledson, M., Majoor, C., Indihar, M., Sawicki, G.S., MacGregor, G., Merkus, P., Keating, C., Daines, C.L., McKone, E.F., Quick, B., Bakker, M., Willey-Courand, D., Mermis, J., Viswanathan, L., Nair, N., Garcia, B., Merlo, C., Brown, C.D., Johannes, J., Indihar, V., Barreto, C., Calimano, F.J., Plant, B., Smith, D.J., Casserly, B.P., Rubenstein, R.C., Eisenmann, S., Tolle, J., Liou, T., Rowe, S.M., Gifford, A.H., Ly, N., Duckers, J., Middleton, P., Heijerman, H., van der Meer, R., Sosnay, P.R., Reyes, S., Ortega, V., Lam, A.P., Nash, E.F., Braun, A., McCoy, K., DiMango, E., Davies, J., Mehdi, N., Peckham, D., McElvaney, N.G., Barrios, C., Tian, S., Tupayachi Ortiz, M., Wilson, J., Fullmer, J., Wang, L.T., Nasr, S., Kiefer, A., Yonker, L., Waltz, D., Mall, M.A., Yonker, L.M., McKone, E., Tullis, E., Schwarz, C., Sutharsan, S., Sheikh, S., Burr, L., Uluer, A.Z., Taylor-Cousar, J.L., Fischer, R., Van Braeckel, E., Klingsberg, R., Solomon, G.M., Ruddy, J., Uluer, A., Jain, R., Pancham, K., Ramsey, B.W., Chu, C., Rosenbluth, D.B., Pilewski, J., Rao, A., Hebestreit, H., Van Goor, F., Billings, J., Horsley, A., Xi, Y., Doe, S., Dunitz, J., Azevedo, P., Griffonnet, J.   +89 more
core   +6 more sources