Results 101 to 110 of about 3,564 (173)

The F508del-CFTR trafficking correctors elexacaftor and tezacaftor are CFTR-independent Ca2+-mobilizing agonists normalizing abnormal Ca2+ levels in human airway epithelial cells

Respiratory Research
Background Cystic fibrosis (CF) is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) channel. For people with CF (pwCF) affected by the most common pathogenic variant F508del, a tritherapy, named ...
Manuella Lévêque, Sandra Mirval, Christine Barrault, Isabelle Fixe, Christelle Coraux, Edouard Sage, Frédéric Becq, Clarisse Vandebrouck   +7 more
doaj   +1 more source

Rethinking hyperbilirubinemia: Gilbert syndrome in children with cystic fibrosis, a case report

Respiratory Medicine Case Reports
Cystic Fibrosis leads to liver complications, including cystic fibrosis liver disease but hyperbilirubinemia in CF patients on CFTR modulators is less understood.
Yara Salameh, John Lyles, Shatha Yousef
doaj   +1 more source

DEVELOPMENT AND VALIDATION OF STABILITY-INDICATING RP-UPLC METHOD FOR THE SIMULTANEOUS ESTIMATION OF TEZACAFTOR AND IVACAFTOR IN FORMULATIONS

, 2020
Objective: Aim of the present research work was to develop a sensitive, rapid and accurate, stability-indicating RP-UPLC method for the simultaneous estimation of tezacaftor and ivacaftor in formulations.
MANEKA, S. LAKSHMI, ANJANA, C. H. K. V. L. S. N., SARAVANAKUMAR, R. T.   +2 more
core  

The long-term effect of elexacaftor/tezacaftor/ivacaftor on cardiorespiratory fitness in adolescent patients with cystic fibrosis: a pilot observational study

BMC Pulmonary Medicine
Background Physical activity is a crucial demand on cystic fibrosis treatment management. The highest value of oxygen uptake (VO2peak) is an appropriate tool to evaluate the physical activity in these patients.
Nela Stastna, Lenka Hrabovska, Pavel Homolka, Lukas Homola, Michal Svoboda, Kristian Brat, Libor Fila   +6 more
doaj   +1 more source

Fetal drug exposure after maternally administered CFTR modulators Elexacaftor/Tezacaftor/Ivacaftor in a rat model

BACKGROUND: The potential effects of the very effective cystic fibrosis triple combination drug, Elexacaftor/Tezacaftor/Ivacaftor (ETI) in pregnancy on prenatal development of offspring remain largely unknown.
Donnelley, M, Habgood, MD, Li, D, Zhu, Y, Schneider-Futschik, EK, Parsons, D   +5 more
core   +2 more sources

Profile of tezacaftor/ivacaftor combination and its potential in the treatment of cystic fibrosis

Therapeutics and Clinical Risk Management, 2019
Dejene Shiferaw,* Shoaib Faruqi*Department of Respiratory Medicine, Hull University Teaching Hospitals NHS Trust, Cottingham HU16 5JQ, UK *These authors contributed equally to this workCorrespondence: Shoaib FaruqiDepartment of Respiratory Medicine ...
Shiferaw D, Faruqi S
doaj  

The expanded French compassionate programme for elexacaftor–tezacaftor–ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study

International audienceBackground Elexacaftor-tezacaftor-ivacaftor has been approved in Europe for people with cystic fibrosis with at least one F508del CFTR variant.
Roy, Charlotte, de Carli, Paola, Richaud-Thiriez, Bénédicte, Montcouquiol, Sylvie, Mayer, Sophie, Audousset, Camille, Mankikian, Julie, Arnouat, Baptiste, Quétant, Sébastien, Audrezet, Marie-Pierre, Verhille, Juliette, Pramil, Stéphanie, Ohlmann, Camille, Campbell, Karine, Hamidfar, Rebecca, Mittaine, Marie, Sermet-Gaudelus, Isabelle, Porzio, Michele, Gachelin, Elsa, Honoré, Isabelle, de Miranda, Sandra, Périsson, Caroline, Socchi, Floriane, Baravalle, Mélisande, Girodon, Emmanuelle, Burnet, Espérie, Llerena, Catherine, Marchal, Sarah, Leroy, Sylvie, Mely, Laurent, Hinzpeter, Alexandre, Blondé, Aurore, Bui, Stéphanie, Coltey, Bérengère, Stremler, Nathalie, Poey, Nora, Grenet, Dominique, Dupuy-Grasset, Magali, da Silva, Jennifer, Gabsi, Asma, Perez, Thierry, Danner-Boucher, Isabelle, Bon, Claire, Chiron, Raphael, Ravoninjatovo, Bruno, Abely, Michel, Foucaud, Pierre, Guillaumot, Anne, Dufeu, Nadine, Kieffer, Sebastien, Nove-Josserand, Raphaële, Belleguic, Chantal, Chedevergne, Frédérique, Fanton, Annlyse, Wizla, Nathalie, Alkoussa, Wael, Cosson, Laure, Durieu, Isabelle, Dalphin, Marie-Laure, Macey, Julie, Murris-Espin, Marlène, Tatopoulos, Aurélie, Perrin, Thomas, Le Rouzic, Olivier, Kanaan, Reem, Duthoit, Louise, Laurans, Muriel, Dehillotte, Clémence, Le Clainche-Viala, Laurence, Nouvel, Thierry, Valois, Sophie, Rames, Cinthia, Thouvenin, Guillaume, Houdouin, Véronique, Reynaud, Quitterie, Corvol, Harriet, Hatton, Aurélie, Deneuville, Eric, Ramel, Sophie, Pranke, Iwona, Priou, Pascaline, Raynal, Caroline, Martin, Clémence, Dupuis, Marion, Burgel, Pierre-Régis, Flament, Thomas, Labbé, Guillaume, Lemonnier, Lydie, Remus, Natascha, Dreano, Elise, Reynaud-Gaubert, Martine, Lebihan, Jean, Marguet, Christophe, Coolen-Allou, Nathalie, Douvry, Benoit, Pouradier, Delphine, Languepin, Jeanne, Scalbert, Manuela, Carlier, Nicolas, Vuillard, Constance, Andrejak, Claire, Weiss, Laurence, Reix, Philippe, Bihouée, Tiphaine   +103 more
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Reduced Use of Maintenance Therapies Among People With Cystic Fibrosis Following Initiation of Elexacaftor/Tezacaftor/Ivacaftor in Australia

PurposeElexacaftor/tezacaftor/ivacaftor (ETI) is an efficacious targeted therapy for cystic fibrosis, but its impact on the use of maintenance therapies has not been assessed in Australia.MethodsWe performed a retrospective cohort study including ...
Jimenez-Solem, Espen, Petersen, Janne, Pearson, Sallie Anne, Jensen, Camilla Bjørn, Falster, Michael O., Litchfield, Melisa, Jaffe, Adam, Råket, Hans Kristian, Wang, Joanna Nan, Milder, Tamara   +9 more
core   +1 more source

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

Decreased pulmonary exacerbations and lower intravenous antibiotic usage following vanzacaftor/tezacaftor/deutivacaftor treatment in people with cystic fibrosis with <i>F508del</i>-minimal function genotypes. [PDF]

Eur Respir J
Horsley A, Arteaga-Solis E, Keating C, Mall MA, Ramsey B, Nair N, Thorat T, Sosnay P.   +7 more
europepmc   +1 more source