Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

Middleton, Peter G., Mall, Marcus A., Dřevínek, Pavel, Lands, Larry C., McKone, Edward F., Polineni, Deepika, Ramsey, Bonnie W., Taylor-Cousar, Jennifer L., Tullis, Elizabeth, Vermeulen, François, Marigowda, Gautham, McKee, Charlotte M., Moskowitz, Samuel M., Nair, Nitin, Savage, Jessica, Simard, Christopher, Tian, Simon, Waltz, David, Xuan, Fengjuan, Rowe, Steven M., Jain, Raksha·2019·New England Journal of Medicine
DOI 10.1056/nejmoa1908639

Cited by (2,225)

Items 1,001–1,100 of 2,225

  1. Decreases in Rhinology Care Utilization by People with Cystic Fibrosis on Highly Effective Modulator Therapy
    Ethan J. Han, Daniel M. Beswick, Patricia H. Eshaghian, Grant A. Turner, Jivianne T. Lee, Douglas A. Li, Marilene B. Wang, Jennifer L. Taylor‐Cousar, Jeffrey D. Suh · 2023 · Annals of Otology Rhinology & Laryngology
  2. One year effect of tezacaftor and ivacaftor on functional exercise capacity and muscle strength in people with cystic fibrosis
    Anne-Sophie Aubriot, Morgane Penelle, Gonçalvès Clémence, Silvia Berardis, Christophe Goubau, Grégory Reychler, Sophie T. Gohy · 2024 · Heliyon
  3. The Changing Landscape of Treatment for Cystic Fibrosis Related Diabetes
    Mehdia Amini, Kevin K. Yu, Jessica Ann Liebich, Vaishaliben Ahir, Emily A. Wood, Stewart Gary Albert, Sandeep Dhindsa · 2024 · Journal of Clinical & Translational Endocrinology
  4. Remote endpoints for clinical trials in cystic fibrosis: Report from the U.S. CF foundation remote endpoints task force
    Jordana E. Hoppe, Jacquelyn Sjoberg, Gina Hong, Katie R. Poch, Edith T. Zemanick, Stephanie A. Thee, Claire L. Edmondson, Dhiren R. Patel, Meghana Sathe, Drucy Borowitz et al. · 2024 · Journal of Cystic Fibrosis
  5. The Role of the Microbiome in Pediatric Respiratory Diseases
    Edith T. Zemanick, Christian Rosas‐Salazar · 2024 · Clinics in Chest Medicine
  6. Adherence to medicine in adolescence
    Christina Joanne Pearce, Aqeem Azam, Peter James Barry, G d’Ancona · 2024 · European Respiratory Society eBooks
  7. Clinical, economic, and societal burden of cystic fibrosis and the impact of the CFTR modulator, lumacaftor/ivacaftor: an assessment using linked registry data in Sweden
    Anders Lindblad, Isabelle de Monestrol, Marita Gilljam, Christina Krantz, Lisa J. McGarry, Jonas Banefelt, Martina Aldvén · 2024 · Journal of Medical Economics
  8. Elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and rare mutations
    Valentina Fainardi, Federico Cresta, Claudio Sorio, Paola Melotti, Emanuela Pesce, Michela Deolmi, Francesco Longo, Karina Kleinfelder, Susanna Esposito, Giovanna Pisi · 2024 · Pediatric Pulmonology
  9. Gene expression profile of intestinal organoids from people with cystic fibrosis upon exposure to elexacaftor/tezacaftor/ivacaftor
    Ondřej Cinek, Eva Fürstová, Stepanka Novotna, Klára Hubáčková, Tereza Doušová, Lucie Bořek-Dohalská, Pavel Dřevı́nek · 2024 · Journal of Cystic Fibrosis
  10. The challenged urine bicarbonate excretion test in cystic fibrosis: A comprehensive analysis of urine acid/base parameters
    Amalie Quist Rousing, Majbritt Jeppesen, Søren Jensen‐Fangel, Jens Leipziger, Mads Vaarby Sørensen, Peder Berg · 2024 · Acta Physiologica
  11. Ivacaftor for Chronic Obstructive Pulmonary Disease: Results from a Phase 2, Randomized Controlled Trial
    Kadambari Vijaykumar, George Marty Solomon, Jennifer Susan Guimbellot, Edward P. Acosta, Pradeep G. Bhambhavni, Sharon White, Harrison Kim, Sammeta Vamsee Raju, Lawrence W. Rasmussen, H. Necole et al. · 2024 · American Journal of Respiratory and Critical Care Medicine
  12. Fatty acid abnormalities in cystic fibrosis–the missing link for a cure?
    Sławomira Drzymała-Czyż, Jarosław Walkowiak, Carla Colombo, Gianfranco Alicandro, Olav Trond Storrøsten, Magnhild L Pollestad Kolsgaard, Egil Bakkeheim, Birgitta Strandvik · 2024 · iScience
  13. Peripheral Muscle Function and Body Composition in People With Cystic Fibrosis on Elexacaftor/Tezacaftor/Ivacaftor: A Cross‐Sectional Single‐Centre Study
    L J, Anthony I. Shepherd, Jo Corbett, Mathieu Gruet, Gary James Connett, Mark Ivan Allenby, Julian P. Legg, Thomas W V Daniels, Don S. Urquhart, ZOE LOUISE SAYNOR · 2025 · Pediatric Pulmonology
  14. Immunogenic adverse events to CFTR modulators – An international survey
    Ruth Maria Urbantat, Laura Behan, Sebastian Wisniewski, Joshua Gardner, Mirjam Stahl, Marcus Alexander Mall, Daniel Gavin Peckham, Dean John Naisbitt, Jobst Fridolin Roehmel · 2025 · Journal of Cystic Fibrosis
  15. Disruption of Polycystin Ciliary Localization and Channel Function by Autosomal Dominant Polycystic Kidney Disease–Causing Polycystin-1 Variants
    Kotdaji Ha, Gabriel B. Loeb, Meyeon Park, Mohona Gupta, Yukako Akiyama, Jillian Argiris, Aide Pinedo, Christine Haewon Park, Nadav Brandes, Fnu Ritu et al. · 2026 · Journal of the American Society of Nephrology
  16. Keep cystic fibrosis patients out of the hospital
    Elliott C. Dasenbrook · 2020 · Cleveland Clinic Journal of Medicine
  17. Therapeutic potential for coxibs-nitric oxide releasing hybrids in cystic fibrosis
    Sara Consalvi, Giovanna Poce, Carla Ghelardini, Lorenzo Di Cesare Mannelli, Paola Patrignani, Annalisa Bruno, Maurizio Anzini, Vincenzo Calderone, Alma Martelli, Lara Testai et al. · 2020 · European Journal of Medicinal Chemistry
  18. Safety of research bronchoscopy with BAL in stable adult patients with cystic fibrosis
    Daniel S Aridgides, John A. Dessaint, Graham T. Atkins, James Carroll, Alix Ashare · 2021 · PLoS ONE
  19. Discrete choice experiment (DCE) to quantify the influence of trial features on the decision to participate in cystic fibrosis (CF) clinical trials
    Rebecca Dobra, Marco Boeri, J.S. Elborn, Frank Kee, Susan L. Madge, Jane Carolyn Davies · 2021 · BMJ Open
  20. Catamenial haemoptysis in females with cystic fibrosis: a case series with review of management strategies
    Kubra Melike Bozkanat, Natalie E. West, Sigrid Ladores, Kristina Montemayor, Maria Gabriela Tupayachi Ortiz, Mindy S. Christianson, Raksha Jain · 2021 · Respirology Case Reports
  21. Clinical Pharmacokinetic and Pharmacodynamic Considerations in the Drug Treatment of Non-Tuberculous Mycobacteria in Cystic Fibrosis
    Andrew Burke, Daniel B. Smith, Chris Coulter, Scott Cameron Bell, Rachel Mary Thomson, Jason Alexander Roberts · 2021 · Clinical Pharmacokinetics
  22. Non-invasive ventilation is associated with long-term improvements in lung function and gas exchange in cystic fibrosis adults with hypercapnic respiratory failure
    L.E. Wadsworth, John Belcher, Rowland J. Bright-Thomas · 2021 · Journal of Cystic Fibrosis
  23. The short-term effects of ORKAMBI (lumacaftor/ivacaftor) on regional and distal lung structures using functional respiratory imaging
    Eline Lauwers, Dennis Belmans, Benjamin Mignot, Kris Ides, Kim Van Hoorenbeeck, Annemiek Snoeckx, Cedric S. Van Holsbeke, Vicky Nowé, Eva Van Braeckel, Wilfried De Backer et al. · 2021 · Therapeutic Advances in Respiratory Disease
  24. Phosphodiesterase 8A Regulates CFTR Activity in Airway Epithelial Cells
    Mark J. Turner, Yukiko Sato, David Y. Thomas, Kathy Abbott-Banner, John W. Hanrahan · 2021 · Cellular Physiology and Biochemistry
  25. Effects of elexacaftor–tezacaftor–ivacaftor discontinuation in cystic fibrosis
    Georgia Mitropoulou, Zisis Balmpouzis, Jérôme Plojoux, Jennifer Dotta-Celio, Alain Sauty, Angela Koutsokera · 2022 · Respiratory Medicine and Research
  26. Non-Modulator Therapies
    Marie E. Egan · 2022 · Clinics in Chest Medicine
  27. 2022 Update of indications and contraindications for lung transplantation in France
    Jérôme Le Pavec, Christophe M. Pison, Sandrine Hirschi, Vincent Bunel, Pierre Mordant, Olivier Brugière, Morgan Le Guen, Anne Olland, Benjamin Coiffard, Benjamin Renaud-Picard et al. · 2022 · Respiratory Medicine and Research
  28. The influence of exocrine pancreatic function on the exposure and pharmacokinetics of ivacaftor in people with cystic fibrosis
    Renske van der Meer, Erik B. Wilms, Margot N Eggermont, Helena M Paalvast, Richard C J M van Rossen, Harry G.M. Heijerman · 2022 · Journal of Cystic Fibrosis
  29. The CFHealthHub Learning Health System: Using Real-Time Adherence Data to Support a Community of Practice to Deliver Continuous Improvement in an Archetypal Long-Term Condition
    Robert David Sandler, Martin James Wildman · 2022 · Healthcare
  30. It Takes Two to Tango! Protein–Protein Interactions behind cAMP-Mediated CFTR Regulation
    Alessandra Murabito, Janki Bhatt, Alessandra Ghigo · 2023 · International Journal of Molecular Sciences
  31. A birth cohort approach to understanding cystic fibrosis lung infections
    Anthony J. Fischer, Paul Joseph Planet · 2023 · Journal of Cystic Fibrosis
  32. Fluorescence assay for simultaneous quantification of CFTR ion-channel function and plasma membrane proximity
    Stella Prins, Emily Langron, Cato Hastings, Emily J. Hill, Andra C. Stefan, Lewis D. Griffin, Paola Vergani · 2020 · Journal of Biological Chemistry
  33. Associations between peak oxygen uptake, lung function, and bronchiectasis in children with cystic fibrosis in the era of CFTR modulators
    Cassidy Du Berry, Nicole Westrupp, Shivanthan Shanthikumar, Liam Welsh · 2021 · Pediatric Pulmonology
  34. Lingering Identity as Chronically Ill and the Unanticipated Effects of Life-Changing Precision Medicine in Cystic Fibrosis: A Case Report
    Sigrid Ladores, Morgan Polen · 2021 · Journal of Patient Experience
  35. Monitoring Neutrophil Elastase and Cathepsin G Activity in Human Sputum Samples
    Dario L. Frey, Matteo Guerra, Marcus Alexander Mall, Carsten Schultz · 2021 · Journal of Visualized Experiments
  36. Lung transplant referrals for individuals with cystic fibrosis: A pediatric perspective on the cystic fibrosis foundation consensus guidelines
    Melinda P. Solomon, George B. Mallory · 2020 · Pediatric Pulmonology
  37. Reply to Kuek et al. : Optimism with Caution: Elexacaftor–Tezacaftor–Ivacaftor in Patients with Advanced Pulmonary Disease
    Pierre‐Régis Burgel, Jennifer Da Silva, Jean-Louis Paillasseur, Clémence Martin · 2021 · American Journal of Respiratory and Critical Care Medicine
  38. CFTR Modulators to the Rescue of Individuals with Cystic Fibrosis and Advanced Lung Disease
    Mike M. Myerburg, Joseph M. Pilewski · 2021 · American Journal of Respiratory and Critical Care Medicine
  39. Respiratory Mycoses: A Call to Action to Recognize, Educate and Invest
    Anand Shah, Darius P. H. Armstrong-James, Sanjay Haresh Chotirmall · 2021 · Mycopathologia
  40. A cell-permeable nanobody to restore F508del cystic fibrosis transmembrane conductance regulator activity
    Luise Franz, Tihomir Rubil, Anita Balázs, Marie Overtus, Kristin Kemnitz‐Hassanin, Cédric Govaerts, Marcus Alexander Mall, Christian P. R. Hackenberger · 2026 · Nature Chemical Biology
  41. The effect of triple CFTR modulator therapy and azithromycin on ion channels and inflammation in cystic fibrosis
    Suhad Bani Melhim, Lisa Douglas, James A Reihill, Damian G. Downey, Lorraine Martin · 2024 · ERJ Open Research
  42. The impact of a whole foods dietary intervention on gastrointestinal symptoms, inflammation, and fecal microbiota in pediatric patients with cystic fibrosis: A pilot study
    Nicole Green, Carson J. Miller, David L. Suskind, Marshall Brown, Christopher E. Pope, Hillary S. Hayden, Sharon McNamara, Anna Kanter, Laura Nay, Lucas R. Hoffman et al. · 2024 · Clinical Nutrition
  43. Mutant protein chemical rescue: From mechanisms to therapeutics
    Timothy R. O’Meara, Brad A. Palanski, Maggie Chen, Yingfeng Qiao, Philip A. Cole · 2025 · Journal of Biological Chemistry
  44. Remote monitoring of cystic fibrosis lung disease in children and young adults
    Marcus Svedberg, Jens Michelsen, Emma Roberts, Huda Abdulahi Ostrand, Christine Rønne Hansen, Christina Krantz, Petrea Ericsson, Ulrika Lindberg, Henrik Imberg · 2025 · Journal of Cystic Fibrosis
  45. Baseline Characteristics and Self‐Reported Use of Chronic Daily Therapies of the Home Reported Outcomes With CFTR Modulator Therapy (HERO‐2) Cohort
    Georgene E. Hergenroeder, Kevin J. Psoter, Cynthia D. Brown, Josh Ostrenga, C. Ford, L. Bendy, Bradley H. Rosen, Nell Meosky Luo, C. Zhang, Kathryn A. Sabadosa et al. · 2025 · Pediatric Pulmonology
  46. CFTR Modulators Counteract F508del CFTR Functional Defects in a Pancreatic Epithelial Model of Cystic Fibrosis
    Alessandra Ludovico, Debora Baroni · 2025 · Life
  47. Severe hepatotoxicity is uncommon following the introduction of Elexacaftor/Tezacaftor/Ivacaftor: A real-world two-years follow-up study of the Danish cystic fibrosis cohort
    Christine Højte, Mette Frahm Olsen, Daniel Faurholt‐Jepsen, Marianne Hørby Jørgensen, Tacjana Pressler, Christian Leo‐Hansen, Thomas Bryrup, Majbritt Jeppesen, Hanne Vebert Olesen, Janne Petersen et al. · 2025 · Journal of Cystic Fibrosis
  48. The clinical, psychosocial, and economic burden of cystic fibrosis lung disease in the era of CFTR modulator therapy
    Isabelle Fajac, Raksha Jain, Marcus Alexander Mall, Bruce K. Rubin, Patrick A. Flume · 2025 · Annals of the American Thoracic Society
  49. Impact of glucagon-like-peptide-1 receptor agonist therapy on pulmonary function in people with cystic fibrosis who achieve normal body mass index
    Andrew M. Horvit, Katie L. Kaput, Amber Neece, Jessica D. Abramowitz, Marconi Abreu, Gregory A. Ratti, James D. Finklea, Raksha Jain, Sasan Mirfakhraee · 2025 · Journal of Cystic Fibrosis
  50. Human induced pluripotent stem cells for in vitro modeling of impaired mucociliary clearance in cystic fibrosis lung disease
    Mark-Christian Klassen, Anita Balázs, Janina Zöllner, Nicole Cleve, Laurien Czichon, Laura von Schledorn, Jan Hegermann, Janna C. Nawroth, Doris Roth, Mia Mielenz et al. · 2025 · Stem Cell Research & Therapy
  51. Real-World Safety and Effectiveness of Elexacaftor, Tezacaftor, and Ivacaftor in People with Cystic Fibrosis and Advanced Lung Disease: A Two-Year Multicenter Cohort Study
    Sonia Volpi, Maura Ambroni, Roberto Buzzetti, Giuseppe G. Cimino, Andrea Gramegna, Maria Cristina Lucanto, Pietro Ripani, Mirco Ros, Donatello Salvatore, Elena Spada et al. · 2025 · International Journal of Molecular Sciences
  52. A Splice Switch in SIGIRR Causes a Defect of IL-37-Dependent Anti-Inflammatory Activity in Cystic Fibrosis Airway Epithelial Cells
    Keiko Ueno-Shuto, Shunsuke Kamei, Megumi Hayashi, Ayami Fukuyama, Yuji Uchida, Naofumi Tokutomi, Mary Ann Suico, Hirofumi Kai, Tsuyoshi Shuto · 2022 · International Journal of Molecular Sciences
  53. Rationale for haploinsufficiency correction therapy in neurofibromatosis type 1
    Michael Frost, Eduard Serra, David H. Viskochil, Bruce R. Korf, Michelle K. Mattson-Hoss, Glenn E. Croston, Herb Sarnoff · 2022 · Journal of Translational Genetics and Genomics
  54. A Novel 7 H ‑[1,2,4]Triazolo[3,4‑ b ]thiadiazine-based Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Directed toward Treatment of Cystic Fibrosis
    András Rab, Yang Xun, William F. Tracy, Jeong Shin Hong, Disha Joshi, Candela Manfredi, S. Ponnaluri, Alexander A. Kolykhalov, Min Qui, Haian Fu et al. · 2023 · ACS Medicinal Chemistry Letters
  55. The impact of elexacaftor/tezacaftor/ivacaftor therapy on the pulmonary management of adults with cystic fibrosis: An expert-based Delphi consensus
    Andrea Gramegna, Stefano Aliberti, Maria Adelaide Calderazzo, Rosaria Casciaro, Clara Ceruti, Giuseppe G. Cimino, Benedetta Fabrizzi, Cristina Lucanto, Barbara Messore, Giovanna Pisi et al. · 2023 · Respiratory Medicine
  56. Preliminary evidence for sustained efficacy of CFTR modulator therapy with concomitant rifabutin administration
    Eunjin Hong, Sarah Parsons, Laura A. Sass, Cynthia Epstein, Lynn Chan, Claire E. Brown, Patricia H. Eshaghian, Paul M. Beringer · 2023 · Journal of Cystic Fibrosis
  57. Putting bicarbonate on the spot: pharmacological insights for CFTR correction in the airway epithelium
    Mirosław Zając, Agathe Lepissier, Elise Dréano, B. Chevalier, Aurélie Hatton, Mairead Kelly, Daniela Guidone, Gabrielle Planelles, Aleksander Edelman, Emmanuelle Girodon et al. · 2023 · Frontiers in Pharmacology
  58. Elexacaftor/tezacaftor/ivacaftor in liver or kidney transplanted people with cystic fibrosis using tacrolimus, a drug-drug interaction study
    Renske van der Meer, Erik B. Wilms, Margot N Eggermont, Helena M Paalvast, Matthijs van Luin, Richard C J M van Rossen, Harry G.M. Heijerman · 2024 · Journal of Cystic Fibrosis
  59. Pharmacokinetic Enhancement of Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis: A Cost Reduction Strategy to Address Global Disparities in Access
    Eunjin Hong, Marco Zampoli, Paul M. Beringer · 2024 · Clinical Pharmacology & Therapeutics
  60. Impact of age at ivacaftor initiation on pulmonary outcomes among people with cystic fibrosis
    Christian A. Merlo, Lisa J. McGarry, Teja Thorat, Catherine Nguyen, Maral DerSarkissian, Aruna Muthukumar, Joe Healy, M. Alan Brookhart, Jaime L. Rubin · 2024 · Thorax
  61. Effect of Modulator Therapies on Nutritional Risk Index in Adults with Cystic Fibrosis: A Prospective Cohort Study
    Nadir Yalçın, Esen Deniz Akman, Oğuz Karcıoğlu, Karel Allegaert, Kutay Demirkan, Ebru Damadoğlu, Alı Fuat Kalyoncu · 2024 · Nutrients
  62. Impact of elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy on rates of endoscopic sinus surgery in cystic fibrosis
    Graham Pingree, Mihai A. Bentan, Thomas H. Fitzpatrick, Theodore A. Schuman · 2024 · International Forum of Allergy & Rhinology
  63. Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis with Mildly Decreased and Normal Lung Function: A Real-Life Observational Study
    Karin Yaacoby‐Bianu, Adi Dagan, Malena Cohen‐Cymberknoh, Lior Tsviban, Michal Gur, Inbal Golan‐Tripto, Micha Aviram, Nili Stein, Michal Shteinberg, Eitan Kerem et al. · 2024 · Annals of the American Thoracic Society
  64. The gastrointestinal microbiome, small bowel bacterial overgrowth, and microbiome modulators in cystic fibrosis
    Nicole Green, Christopher Chan, Chee Yee Ooi · 2024 · Pediatric Pulmonology
  65. Effect of CFTR Modulators on Oxidative Stress and Autophagy in Non-CFTR-Expressing Cells
    Filippo Scialò, Gustavo Cernera, Lorenza Polise, Giuseppe Castaldo, Felice Amato, Valeria Rachela Villella · 2024 · International Journal of Molecular Sciences
  66. Immune-Mediated Megaconial Myopathy
    Ashley R. Santilli, Oliver Ni, Margherita Milone, Duygu Selcen, Anahit Mehrabyan, Arjun Seth, Christine Hsieh, Wasim Fawzi Raslan, Mohammed Khalid Alkhalifah, Raed M. Alenezi et al. · 2024 · Neurology
  67. Pharmacological rescue of the G85E CFTR variant by preclinical and approved modulators
    Valeria Tomati, Valeria Capurro, Emanuela Pesce, Cristina Pastorino, Elvira Sondo, Mariateresa Lena, Anna Borrelli, Federico Cresta, Stefano Pantano, Francesca Collini et al. · 2024 · Frontiers in Pharmacology
  68. Testing organ-specific responses to therapies in tissues differentiated from Cystic Fibrosis patient derived iPSCs
    Abdelkader Daoud, Sunny Xia, Onofrio Laselva, Janet X. Jiang, Christine E. Bear · 2025 · Stem Cell Research
  69. Evaluation of Elexacafor/Tezacaftor/Ivacaftor therapy after lung transplantation in Cystic Fibrosis: The Dutch National KOALA study
    Johanna P. van Gemert, Bart Luijk, Merel E. Hellemons, K.A. Visser, Carina M.E. Hansen, Renske van der Meer, C. Tji Gan, Hester van der Vaart, Onno W. Akkerman, Willie N. Steenhuis et al. · 2025 · JHLT Open
  70. Short-term modification of breathprint by Elexacaftor/Tezacaftor/Ivacaftor in a paediatric cohort
    Emmanuelle Bardin, Nicolas Hunzinger, Elodie Lamy, Camille Roquencourt, Bingqing Zhou, Y. Tabache, Laurence Le Clainche, Natascha Remus, Charlotte Roy, Philippe Devillier et al. · 2025 · Journal of Cystic Fibrosis
  71. Real-life impact of genotype and severity of lung disease on efficacy of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis
    Shahid Ijaz Sheikh, Melissa Holtzlander, Mariah Eisner, Courtney A. Gushue, SABRINA K PALACIOS, Kavitha S. Kotha, Sehyr Imran, Karen McCoy · 2025 · Pulmonary Pharmacology & Therapeutics
  72. The impact of pregnancy on mortality and lung function in cystic fibrosis patients
    Paul K. Mohabir, Fatma Güntürkün, Jennifer Cannon, Yaowei Deng, Ariadna García, Eahsan Shahriary, Alicia A. Mirza · 2025 · Journal of Cystic Fibrosis
  73. Change in the 6-min walk test among 71 patients with cystic fibrosis treated with elexacaftor/tezacaftor/ivacaftor
    Julien Perrone, Muriel Rabilloud, Laurent Mély · 2025 · Respiratory Medicine
  74. Cannabinoid receptor 2 agonist, lenabasum, for the treatment of pulmonary exacerbations in cystic fibrosis
    Natalie E. West, Michael W. Konstan, Patrick A. Flume, Donald R. VanDevanter, Amalia Magaret, Henryk Mazurek, E. Amelina, István Laki, R. Chiron, Sivagurunathan Sutharsan et al. · 2025 · Journal of Cystic Fibrosis
  75. Overcoming Immunological Challenges to Helper-Dependent Adenoviral Vector-Mediated Long-Term CFTR Expression in Mouse Airways
    Huibi Cao, Rongqi Cathleen Duan, Jim Hu · 2020 · Genes
  76. Aerosolized lancovutide in adolescents (≥12 years) and adults with cystic fibrosis – a randomized trial
    Ernst Eber, Maria Trawinska-Bartnicka, Dorota Sands, G. Bellon, Uwe Mellies, Katalin Bolbas, Serena Quattrucci, Henryk Mazurek, Rudolf S. Widmann, Christian Schoergenhofer et al. · 2020 · Journal of Cystic Fibrosis
  77. A formalized transition program for cystic fibrosis: A 10‐year retrospective analysis of 97 patients in Lyon
    Gaëlle Bourgeois, Fanny Magne, Raphaele Nove Josserand, Stéphane Durupt, I. Durieu, Philippe Reix, Quitterie Reynaud · 2021 · Pediatric Pulmonology
  78. “Il faut continuer à poser des questions” patient reported outcome measures in cystic fibrosis: An anthropological perspective
    R. Coucke, Audrey Chansard, Véronique Bontemps, Dominique Grenet, D. Hubert, Clémence Martin, Elise J. Lammertyn, Emmanuelle Bardin, Veerle Bulteel, F. Chedevergne et al. · 2021 · Journal of Cystic Fibrosis
  79. A new path for CF clinical trials through the use of historical controls
    Amalia Magaret, Mark N. Warden, Noah Simon, Sonya L. Heltshe, George Z. Retsch‐Bogart, Bonnie W. Ramsey, Nicole Mayer-Hamblett · 2021 · Journal of Cystic Fibrosis
  80. The Canadian Glomerulonephritis Registry (CGNR) and Translational Research Initiative: Rationale and Clinical Research Protocol
    Ainslie M. Hildebrand, Moumita Barua, Sean J. Barbour, Karthik Tennankore, Daniel C. Cattran, Tomoko Takano, Ping Lam, Sacha A. De Serres, Ratna Samanta, Michelle Hladunewich et al. · 2022 · Canadian Journal of Kidney Health and Disease
  81. Diagnosis of cystic fibrosis in adulthood and eligibility for novel CFTR modulator therapy
    Hannah J. Farley, Sarah Poole, S. J. Chapman, William George Flight · 2021 · Postgraduate Medical Journal
  82. CFTR dysfunction and targeted therapies: A vision from non-cystic fibrosis bronchiectasis and COPD
    Miguel Ángel Martínez‐García, Jesús María Sierra-Párraga, Esther Quintana, José Luís López-Campos · 2022 · Journal of Cystic Fibrosis
  83. Treatment With LAU-7b Complements CFTR Modulator Therapy by Improving Lung Physiology and Normalizing Lipid Imbalance Associated With CF Lung Disease
    Amanda Centorame, Daciana Catalina Dumut, Mina Youssef, Martin Ondra, Irenej Kianicka, Juhi Shah, Radu Alexandru Paun, Tomáš Oždian, John W. Hanrahan, Ekaterina Gusev et al. · 2022 · Frontiers in Pharmacology
  84. Discovery and SAR of 4-aminopyrrolidine-2-carboxylic acid correctors of CFTR for the treatment of cystic fibrosis
    Marc J. C. Scanio, Xenia B. Searle, Bo Liu, John R. Koenig, Robert J. Altenbach, Gregory A. Gfesser, Andrew R. Bogdan, Stephen N. Greszler, Gang Zhao, Ashvani K. Singh et al. · 2022 · Bioorganic & Medicinal Chemistry Letters
  85. Emerging medicines to improve the basic defect in cystic fibrosis
    Isabelle Fajac, Isabelle Sermet‐Gaudelus · 2022 · Expert Opinion on Emerging Drugs
  86. Hemoptysis and the Risk for Lung Transplant or Death without Transplant in Individuals with Cystic Fibrosis in the United States
    Omar F. Bayomy, Kathleen J. Ramos, Travis Hee Wai, Siddhartha G. Kapnadak, Eric D. Morrell, J. Treadway Nomitch, Lauren R. Pollack, Erika D. Lease, Moira L. Aitken, Anne L. Stephenson et al. · 2022 · Annals of the American Thoracic Society
  87. Time to change course and tackle CF related obesity
    L.R. Caley, Daniel Gavin Peckham · 2022 · Journal of Cystic Fibrosis
  88. Pharmacological chaperone-rescued cystic fibrosis CFTR-F508del mutant overcomes PRAF2-gated access to endoplasmic reticulum exit sites
    Kusumika Saha, B. Chevalier, Stéphane Doly, Nesrine Baatallah, Thomas Guilbert, Iwona Maria Pranke, Mark G. H. Scott, Hervé Enslen, Ida Chiara Guerrera, Cérina Chuon et al. · 2022 · Cellular and Molecular Life Sciences
  89. Drug treatment of cystic fibrosis
    Matthew Bruorton, Thomas R. Goddard · 2022 · Australian Prescriber
  90. Elexacaftor/tezacaftor/ivacaftor—real-world clinical effectiveness and safety. A single-center Portuguese study
    Kelly Lopes, Catarina Custódio, Carlos Lopes, Raquel Bolas, Pilar Azevedo · 2023 · Jornal Brasileiro de Pneumologia
  91. Survey of exercise testing and training in cystic fibrosis clinics in the UK: a decade of progress
    Owen W. Tomlinson, ZOE LOUISE SAYNOR, Daniel J. Stevens, Joe Antoun, Don S. Urquhart, Craig Anthony Williams · 2023 · BMJ Open
  92. Digital technology for delivering and monitoring exercise programs for people with cystic fibrosis
    Ana Carolina Pereira Nunes Pinto, Sara Regina Piva, Aline Pereira da Rocha, Mansueto Gomes Neto, Álvaro Nagib Atallah, Humberto Saconato, Virgínia Fernandes Moça Trevisani · 2023 · Cochrane Database of Systematic Reviews
  93. Nonsense mutations accelerate lung disease and decrease survival of cystic fibrosis children
    Annalisa Orenti, Iwona Maria Pranke, Caroline Faucon, Jessica Varilh, Aurélie Hatton, Anita Golec, Clémence Dehillotte, I. Durieu, Philippe Reix, Pierre‐Régis Burgel et al. · 2023 · Journal of Cystic Fibrosis
  94. “We’ll deal with it as it comes”: A qualitative analysis of romantic partners’ dyadic coping in cystic fibrosis
    Nancy Lau, Kathleen J. Ramos, Moira L. Aitken, Christopher Hooper Goss, Krysta S. Barton, Erin K. Kross, Ruth A. Engelberg · 2023 · Journal of Social and Personal Relationships
  95. Development and validation of a novel personalized electronic patient-reported outcome measure to assess quality of life (Q-LIFE): a prospective observational study in people with Cystic Fibrosis
    Danya Muilwijk, Tessa J. van Paridon, Doris C. van der Heijden, Brenda M. Faber-Bisschop, Domenique D. Zomer-van Ommen, Harry G.M. Heijerman, Cornelis Korstiaan van der Ent · 2023 · EClinicalMedicine
  96. Mathematical models of cystic fibrosis as a systemic disease
    Daniel V. Olivença, Jacob D. Davis, Carla M. Kumbale, Conan Zhao, Sam P. Brown, Nael A. McCarty, Eberhard O. Voit · 2023 · WIREs Mechanisms of Disease
  97. Prospective evaluation of nontuberculous mycobacteria disease in cystic fibrosis: The design of the PREDICT study
    Stacey L. Martiniano, Silvia M. Caceres, Katie R. Poch, N. Rysavy, Valerie K. Lovell, Emily Armantrout, Marion C. Jones, Margaret Anthony, Allison Keck, David P. Nichols et al. · 2023 · Journal of Cystic Fibrosis
  98. Olfactory loss in people with cystic fibrosis: Community perceptions and impact
    Jessa E. Miller, Christine M. Liu, Edith T. Zemanick, Jason C. Woods, Christopher Hooper Goss, Jennifer L. Taylor‐Cousar, Daniel M. Beswick · 2023 · Journal of Cystic Fibrosis
  99. A longitudinal analysis of respiratory symptoms in people with cystic fibrosis with advanced lung disease on and off ETI
    Eliana R Gill, Lauren Bartlett, T. Milinic, Nora Burdis, Joseph M. Pilewski, Jordan Dunitz, Siddhartha G. Kapnadak, Christopher Hooper Goss, Kathleen J. Ramos · 2023 · Journal of Cystic Fibrosis
  100. Synthesis and Evaluation of Ivacaftor Derivatives with Reduced Lipophilicity
    Melissa Iazzi, Phillip Junor, Jitesh Doshi, Saujanya Acharya, Roxana Sühring, Russell D. Viirre, Gagan D. Gupta · 2023 · ACS Omega