Results 21 to 30 of about 1,459,532 (261)

Validation of nasospheroids to assay CFTR functionality and modulator responses in cystic fibrosis [PDF]

Scientific Reports, 2021
The availability of a simple, robust and non-invasive in vitro airway model would be useful to study the functionality of the cystic fibrosis transmembrane regulator (CFTR) protein and to personalize modulator therapy for cystic fibrosis (CF) patients ...
Maite Calucho, Silvia Gartner, Paula Barranco, Paula Fernández-Álvarez, Raquel García Pérez, Eduardo F. Tizzano   +5 more
doaj   +2 more sources

Changing profile of bacterial infection and microbiome in cystic fibrosis: when to use antibiotics in the era of CFTR-modulator therapy. [PDF]

Eur Respir Rev
The advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy, especially the triple therapy combining the drugs elexacaftor, tezacaftor, ivacaftor (ETI), has significantly changed the course of the disease in people with ...
Milczewska J, Syunyaeva Z, Żabińska-Jaroń A, Sands D, Thee S.   +4 more
europepmc   +2 more sources

Heterogeneity of CFTR modulator-induced sweat chloride concentrations in people with cystic fibrosis. [PDF]

J Cyst Fibros
Background: Sweat chloride (SC) concentrations in people with cystic fibrosis (PwCF) reflect relative CF transmembrane conductance regulator (CFTR) protein function, the primary CF defect.
Zemanick ET, Emerman I, McCreary M, Mayer-Hamblett N, Warden MN, Odem-Davis K, VanDevanter DR, Ren CL, Young J, Konstan MW, CHEC-SC Study Group.   +10 more
europepmc   +2 more sources

Caution advised in the use of CFTR modulator treatment for individuals harboring specific CFTR variants

Journal of Cystic Fibrosis, 2022
In December 2020, the U.S. Food and Drug Administration (FDA) expanded the list of CFTR variants approved for treatment with CFTR modulators drugs from 39 to 183.
Karen S Raraigh, , Mary Corey
exaly   +2 more sources

Impact of CFTR modulator therapy on body composition as assessed by thoracic computed tomography: A follow-up study

Nutrition
OBJECTIVE Treatment with cystic fibrosis transmembrane conductance regulator (CFTR) modulators in individuals with cystic fibrosis (CF) has brought a significant change in forced expiratory volume in 1 second (FEV1) and clinical parameters.
VÍCTOR Navas-Moreno, Fernando Sebastián-Vallés, VÍCTOR Rodriguez-Laval   +2 more
exaly   +2 more sources

“CFTR Modulator Theratyping: Current Status, Gaps and Future Directions”

Journal of Cystic Fibrosis, 2018
Background New drugs that improve the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein with discreet disease-causing variants have been successfully developed for cystic fibrosis (CF) patients.
J. Clancy, C. Cotton, S. Donaldson, G. Solomon, D. VanDevanter, M. Boyle, M. Gentzsch, J. Nick, B. Illek, J. Wallenburg, E. Sorscher, M. Amaral, J. Beekman, A. Naren, R. Bridges, P. Thomas, G. Cutting, S. Rowe, A. Durmowicz, M. Mense, K. Boeck, W. Skach, C. Penland, Bruce C. Marshall, A. Faro, E. Joseloff, H. Bihler, J. Mahoney, D. Borowitz, K. Tuggle   +29 more
semanticscholar   +2 more sources

Long-term effectiveness of dual CFTR modulator treatment of cystic fibrosis [PDF]

ERJ Open Research, 2022
Background Although short-term efficacy of lumacaftor/ivacaftor and tezacaftor/ivacaftor is clearly established in clinical trials, data on long-term effectiveness is limited.
D. Muilwijk, D. Z. Zomer-van Ommen, V. Gulmans, M. Eijkemans, C. van der Ent, J. S.W.J. H.G.M. K.M. M. R.A.S. H.M. R. M. H. G.H. L. Altenburg Terheggen-Lagro Heijerman de Winter-de G, J. Altenburg, S. Terheggen‐Lagro, H. Heijerman, K. M. de Winter-de Groot, M. Bakker, R. Hoek, H. Janssens, R. van der Meer, M. Nuijsink, H. van der Vaart, G. Koppelman, L. Conemans, M. Bannier, J. Noordhoek   +19 more
semanticscholar   +2 more sources

CFTR modulator use in post lung transplant recipients

Journal of Heart and Lung Transplantation, 2021
Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapy has previously been contraindicated in solid organ transplant recipients. This was due to lack of data and concern for interactions with immunosuppressive drug regimens. However,
Lauryn A Benninger, Cesar Trillo-Alvarez, Jorge Lascano   +2 more
exaly   +2 more sources

Eligibility of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapies: cohort of cystic fibrosis registry of Türkiye

The Turkish Journal of Pediatrics
Background. Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) variants are essential for determining eligibility for CFTR modulator drugs (CFTRms).
Meltem Akgül Erdal, Halime Nayır Büyükşahin, Velat Şen, Ayşe Ayzıt Kılınç, Haluk Çokuğraş, Güzide Doğan, Aslı İmran Yılmaz, Gökçen Ünal, Mahir Serbes, Derya Ufuk Altıntaş, Elif Arık, Özlem Keskin, Mehmet Mustafa Özaslan, Oğuz Karcıoğlu, Mehmet Köse, Abdurrahman Erdem Başaran, Eylül Pınar Çakır, Yakup Canıtez, Ali Özdemir, Koray Harmancı, Salih Uytun, Sanem Eryılmaz Polat, Melih Hangül, Hasan Yüksel, Gizem Özcan, Pervin Korkmaz, Mehmet Kılıç, Zeynep Gökçe Gayretli Aydın, Gönül Çaltepe, Demet Can, Sibel Doğru, Gökçen Kartal Öztürk, Ayşe Süleyman, Erdem Topal, Beste Özsezen, Mina Hızal, Ezgi Demirdöğen, Hamza Ogun, Şermin Börekçi, Hakan Yazan, Hadice Selimoğlu Şen, Ayşegül Doğan Demir, Erkan Çakır, Tuğba Şişmanlar Eyüboğlu, Nagehan Emiralioğlu, Sevgi Pekcan, Uğur Özçelik, Deniz Doğru   +47 more
doaj   +5 more sources

Different CFTR modulator combinations downregulate inflammation differently in cystic fibrosis [PDF]

eLife, 2020
Previously, we showed that serum and monocytes from patients with CF exhibit an enhanced NLRP3-inflammasome signature with increased IL-18, IL-1β, caspase-1 activity and ASC speck release (Scambler et al. eLife 2019).
H. Jarosz-Griffiths, T. Scambler, C. Wong, S. Lara-Reyna, J. Holbrook, F. Martinon, S. Savic, P. Whitaker, C. Etherington, G. Spoletini, I. Clifton, A. Mehta, M. McDermott, D. Peckham   +13 more
semanticscholar   +2 more sources