Results 31 to 40 of about 3,564 (173)

Physiologically Based Pharmacokinetic Modeling of CFTR Modulation in People with Cystic Fibrosis Transitioning from Mono or Dual Regimens to Triple-Combination Elexacaftor/Tezacaftor/Ivacaftor

Pulmonary Therapy, 2020
Introduction The triple-combination (TC) cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen elexacaftor, tezacaftor, and ivacaftor was shown to be safe and efficacious in phase 3 trials of people with cystic fibrosis (pwCF) ≥ 12 
Alice Tsai, Shu-Pei Wu, Eric Haseltine, Sanjeev Kumar, Samuel M. Moskowitz, Paul Panorchan, Kushal Shah   +6 more
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Regulatory T cell enhancement in adults with cystic fibrosis receiving Elexacaftor/Tezacaftor/Ivacaftor therapy

Frontiers in Immunology, 2023
IntroductionCystic fibrosis (CF), especially CF lung disease, is characterized by chronic infection, immune dysfunction including impairment of regulatory T cells (Tregs) and an exaggerated inflammatory response.
Dirk Westhölter, Jonas Raspe, Hendrik Uebner, Johannes Pipping, Mona Schmitz, Svenja Straßburg, Sivagurunathan Sutharsan, Matthias Welsner, Christian Taube, Sebastian Reuter   +9 more
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DataSheet3_Changes in cystic fibrosis transmembrane conductance regulator protein expression prior to and during elexacaftor-tezacaftor-ivacaftor therapy.PDF

, 2023
Background: Defects in expression, maturation or function of the epithelial membrane glycoprotein CFTR are causative for the progressive disease cystic fibrosis.
Frauke Stanke (32239), Gesine Hansen (194515), Burkhard Tümmler (4273), Rebecca Minso (14512229), Sibylle Junge (494286), Anna-Maria Dittrich (306619), Ella M. Eichhorn (14512226), Silke Hedtfeld (14512223), Sophia T. Pallenberg (14512220), Annette Sauer-Heilborn (691740), Stephanie Tamm (117858), Tobias Welte (87188), Felix C. Ringshausen (14512232)   +12 more
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Personalized Selection of a CFTR Modulator for a Patient with a Complex Allele [L467F;F508del]

Current Issues in Molecular Biology, 2022
The presence of complex alleles in the CFTR gene can lead to difficulties in diagnosing cystic fibrosis and cause resistance to therapy with CFTR modulators.
Elena Kondratyeva, Nataliya Bulatenko, Yuliya Melyanovskaya, Anna Efremova, Elena Zhekaite, Viktoriya Sherman, Anna Voronkova, Irina Asherova, Alexander Polyakov, Tagui Adyan, Valeriia Kovalskaia, Tatiana Bukharova, Dmitry Goldshtein, Sergey Kutsev   +13 more
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DataSheet2_Changes in cystic fibrosis transmembrane conductance regulator protein expression prior to and during elexacaftor-tezacaftor-ivacaftor therapy.PDF

, 2023
Background: Defects in expression, maturation or function of the epithelial membrane glycoprotein CFTR are causative for the progressive disease cystic fibrosis.
Frauke Stanke (32239), Gesine Hansen (194515), Burkhard Tümmler (4273), Rebecca Minso (14512229), Sibylle Junge (494286), Anna-Maria Dittrich (306619), Ella M. Eichhorn (14512226), Silke Hedtfeld (14512223), Sophia T. Pallenberg (14512220), Annette Sauer-Heilborn (691740), Stephanie Tamm (117858), Tobias Welte (87188), Felix C. Ringshausen (14512232)   +12 more
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Vanzacaftor–tezacaftor–deutivacaftor versus elexacaftor–tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103) : results from two randomised, active-controlled, phase 3 trials [PDF]

BackgroundThe goal of cystic fibrosis transmembrane conductance regulator (CFTR) modulators is to reach normal CFTR function in people with cystic fibrosis.
Fabrizzi, Benedetta, Mazurek, Henryk, Weiner, Daniel, Frederick, Carla, Calimano, Francisco, Lorenz, Michael, Sands, Dorota, Duckers, Jamie, Lam, Anna, Wilhelm, Andrew, Carroll, Mary, Fortner, Christopher, Drevinek, Pavel, Atkinson, Jeffrey, Gohy, Sophie, Jain, Raksha, Singer, Jonathan, Sermet-Gaudelus, Isabelle, Williamson, Michael, Tullis, Elizabeth, Uluer, Ahmet, Ramsey, Bonnie, Simmonds, Nicholas, Krick, Stefanie, Withers, Nicholas, Eber, Ernst, McKone, Edward, Mulrennan, Siobhain, Ahluwalia, Neil, Asensio de la Cruz, Oscar, Hoppe, Jordana, Clements, Barry, Ruddy, Jennifer, Mall, Marcus A, Owens, Louisa, Green, Deanna, Vermeulen, François, Froh, Deborah, Robinson, Philip, Waller, Michael, Staples, Heather, Moffett, Kathryn, Liou, Theodore, Mehdi, Nighat, Tremblay, Francois, Welter, John, Legg, Julian, Dacco, Valeria, O'Carroll, Mark, Linnemann, Rachel W, VX20-121-102 Study Group, [missing], Keens, Thomas, Johnson, Larry, Livingston, Floyd, Daines, Cori, Doe, Simon, Ledson, Martin, Studnicka, Michael, Elnazir, Basil, Peckham, Daniel, Bui, Stephanie, Vanderhelst, Eef, Brown, Cynthia, Olesen, Hanne, Zahigian, Rachel, Quintana-Gallego, Esther, Houdouin, Veronique, Mermis, Joel, Plant, Barry, Billings, Joanne, Trillo-Alvarez, Cesar, van der Meer, Renske, Urquhart, Don, Wang, Janice, Ren, Clement, O'Sullivan, Brian, Messore, Barbara, Haworth, Charles, Lands, Larry, Millard, Susan, Le Rouzic, Olivier, Stahl, Mirjam, Nair, Nitin, Mukadam, Zubin, Deschamps, Ashley, Horsley, Alexander, Linnane, Barry, Sutharsan, Sivagurunathan, Parkins, Michael, Appelt, Dorothea, Yonker, Lael, Altenburg, Josje, Tupayachi Ortiz, Maria Gabriela, Epaud, Ralph, Fischer Biner, Reta, Ramel, Sophie, Bakkeheim, Egil, Hilliard, Tom, Pressler, Tacjana, Brown, Sarah, Gartner, Silvia, Callison, John, Cohen-Cymberknoh, Malena, Holliday, Zachary, Marguet, Christophe, Ringshausen, Felix, Byrnes, Catherine, Cox, Desmond, Middleton, Peter, Egan, Marie, Hebestreit, Helge, Lambert, Allison, Knoop, Christiane, Cols Roig, Maria, Burr, Lucy, Mueller, Gary, Casserly, Brian, Linnemann, Rachel, Reix, Philippe, Marion, Chad, Yonker, Lael M, Goss, Christopher, Lee, Timothy, Dubin, Patricia, Wainwright, Claire, Mankikian, Julie, Schaeffer, David, Martin, Hannah, Forseen, Caralee, Blitz Castro, Enrique, Barbosa, Telma, Tolle, James, Gleiber, Wolfgang, Epton, Michael, Luna, Carmen, Salvatore, Donatello, Lindberg, Ulrika, Tirakitsoontorn, Pornchai, Sauty, Alain, Klingsberg, Ross, Michelson, Peter, Quick, Bryon, McCoy, Karen, Teneback, Charlotte, Schwarz, Carsten, McKone, Edward F, Roberts, Jon, Sosnay, Patrick R, Leroy, Sylvie, Wurth, Mark, Quon, Bradley, Manika, Katerina, Dillenhofer, Stefanie, Kremer, Ted, Lapey, Allen, Nicholson, John Michael, Kennedy, John, Henderson, Daniel, Koser, Usma, VX20-121-103 Study Group, [missing], Grenet, Dominique, Sears, Edmund, Ruiz, Fadel, Laki, Istvan, Chiron, Raphael, Efrati, Ori, van der Ent, Kors, Bilodeau, Lara, Braun, Andrew, Elidemir, Okan, Braun, Andrew T, Poplawska, Krystyna, Lopez Neyra, Alejandro, Schellhase, Dennis, Desai, Maya, Naehrig, Susanne, Homola, Lukas, Gramegna, Andrea, Ruppel, Renate, Fiel, Stanley, King, Christopher, Sapiejka, Ewa, Chmiel, James, Roukema, Jolt, Cipolli, Marco, De Monestrol, Isabelle, Omlor, Gregory, Engan, Mette, Johannes, Jimmy, Maitra, Anirban, Kotsimbos, Tom, Filbrun, Amy, DiMango, Emily, Durieu, Isabelle, Taccetti, Giovanni, Stehling, Florian, Fischer, Rainald, Lysinger, Jerimiah, Mondejar-Lopez, Pedro, Kherani, Tamizan, Pancham, Krishna, Fajac, Isabelle, Floreth, Tim, Keating, Claire, Amorim, Adelina, Kamphuis, Lieke, Scher, Herschel, Chatziagorou, Elpis, Wong, Janice, Tissot, Adrien, Wallace, James, Trimble, Aaron, Barrios, Christopher, Gilljam, Marita, Sole Jover, Amparo, Jain, Manu, Bagheri-Potthoff, Azadeh, Castellani, Carlo, Moeller, Alexander, Taylor-Cousar, Jennifer, Jia, Shijing, Escobar, Hugo, Szepfalusi, Zsolt, Sivam, Sheila, Azevedo, Pilar, Bode, Sebastian, Patel, Neil, Halasz, Adrien, Chilvers, Mark, Prais, Dario, Reyes, Santiago, Barry, Peter, Brown, Perry, Tatopoulos, Aurelie, Van Braeckel, EvaGE35UZGent0019990642098020000443910000-0002-7242-0747F85E6FEA-F0ED-11E1-A9DE-61C894A0A6B4, Taylor-Cousar, Jennifer L, Weiss, Laurence, Shkolnik, Boris, Dittrich, Anna-Maria, Morrissey, Brian, MacGregor, Gordon   +240 more
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Combined CFTR modulator therapies are linked with anabolic benefits and insulin-sparing in cystic fibrosis-related diabetes

Journal of Clinical & Translational Endocrinology, 2023
Aims: Combined CFTR modulator therapies have dramatically altered pulmonary outcomes in patients with cystic fibrosis (CF). Their impact on glucose metabolism requires further investigations.
Fabian Lurquin, Sophie Gohy, Michel P. Hermans, Vanessa Preumont   +3 more
doaj   +1 more source

DataSheet1_Elexacaftor-Tezacaftor-Ivacaftor Treatment Reduces Abdominal Symptoms in Cystic Fibrosis-Early results Obtained With the CF-Specific CFAbd-Score.docx

, 2022
Background: The novel and highly effective CFTR modulator combination of elexacaftor-tezacaftor-ivacaftor (ETI) has been shown to improve lung function and body weight in people with Cystic Fibrosis (pwCF) carrying a F508del mutation. However, the impact
Lilith Bechinger (12749372), Patience Eschenhagen (8959820), Laura Caley (12749381), Lenny Sasse, Carsten Schwarz (3161535), Laura Caley, Carlos Zagoya (12749360), Ludwik Kurzidim (12749378), Franziska Duckstein (12749375), Lutz Naehrlich (389423), Carlos Zagoya, Lilith Bechinger, Patience Eschenhagen, Ludwik Kurzidim, Jochen G. Mainz (12749357), Franziska Duckstein, Carsten Schwarz, Olaf Eickmeier, Louise Polte (12749363), Jochen G. Mainz, Daniel Peckham (1687663), Anton Barucha (12749369), Olaf Eickmeier (205876), Christina Smaczny, Daniel Peckham, Christina Smaczny (494287), Lutz Naehrlich, Louise Polte, Lenny Sasse (12749366), Anton Barucha   +29 more
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DataSheet1_Qualitative and quantitative evaluation of computed tomography changes in adults with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor: a retrospective observational study.docx

, 2023
Introduction: The availability of highly effective triple cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapy with elexacaftor–tezacaftor–ivacaftor (ETI) has improved pulmonary outcomes and quality of life of people ...
Frank Wacker (281957), Oliver Weinheimer, Oliver Lammers, Mark O. Wielpütz (8600322), Jan Fuge (532257), Annette Sauer-Heilborn, Sabine Dettmer (549056), Oliver Lammers (17020161), Frank Wacker, Oliver Weinheimer (448627), Sabine Dettmer, Felix C. Ringshausen, Mark O. Wielpütz, Annette Sauer-Heilborn (691740), Tobias Welte, Tobias Welte (87188), Jan Fuge, Felix C. Ringshausen (14512232)   +17 more
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Different CFTR modulator combinations downregulate inflammation differently in cystic fibrosis

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).
Heledd H Jarosz-Griffiths, Thomas Scambler, Chi H Wong, Samuel Lara-Reyna, Jonathan Holbrook, Fabio Martinon, Sinisa Savic, Paul Whitaker, Christine Etherington, Giulia Spoletini, Ian Clifton, Anil Mehta, Michael F McDermott, Daniel Peckham   +13 more
doaj   +1 more source