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

Cited by (2,225)

Items 201–300 of 2,225

  1. An Update on CFTR Modulators as New Therapies for Cystic Fibrosis
    John A. King, Anna-Louise Nichols, Siân Bentley, S.B. Carr, Jane Carolyn Davies · 2022 · Pediatric Drugs
  2. Effects of a Partially Supervised Conditioning Program in Cystic Fibrosis: An International Multicenter, Randomized Controlled Trial (ACTIVATE-CF)
    Helge U. Hebestreit, Susi Kriemler, Christian Schindler, Lothar Stein, Chantal Karila, Don S. Urquhart, David M. Orenstein, Larry C. Lands, Jonathan Schaeff, Ernst Eber et al. · 2021 · American Journal of Respiratory and Critical Care Medicine
  3. Short-Term Effects of Elexacaftor/Tezacaftor/Ivacaftor Combination on Glucose Tolerance in Young People With Cystic Fibrosis—An Observational Pilot Study
    Insa Korten, Elisabeth Kieninger, Linn Krueger, Marina Bullo, Christa E. Flück, Philipp Latzin, Carmen Casaulta, Claudia Boettcher · 2022 · Frontiers in Pediatrics
  4. Sweat chloride reflects CFTR function and correlates with clinical outcomes following CFTR modulator treatment
    Edith T. Zemanick, Bonnie W. Ramsey, Dorota Sands, Edward F. McKone, Isabelle Fajac, Jennifer L. Taylor‐Cousar, Marcus Alexander Mall, Michael W. Konstan, Nitin Nair, Jiaqiang Zhu et al. · 2025 · Journal of Cystic Fibrosis
  5. Effect of highly effective modulator therapy on quality of life in adults with cystic fibrosis
    Emily DiMango, Daniel B. Spielman, Jonathan B. Overdevest, Claire L. Keating, Sarah Fracasso Francis, David Dansky, David A. Gudis · 2020 · International Forum of Allergy & Rhinology
  6. Rethinking physical exercise training in the modern era of cystic fibrosis: A step towards optimising short-term efficacy and long-term engagement
    Mathieu Gruet, ZOE LOUISE SAYNOR, Don S. Urquhart, Thomas Radtke · 2021 · Journal of Cystic Fibrosis
  7. Treatment Preference Among People With Cystic Fibrosis
    Rory A. Cameron, Daniel Office, Jessie Matthews, Mark Rowley, Janice Abbott, Nicholas J. Simmonds, Jennifer A. Whitty, S.B. Carr · 2022 · CHEST Journal
  8. CFTR Modulators: Current Status and Evolving Knowledge
    Lucile Regard, Clémence Martin, Jennifer Da Silva, Pierre‐Régis Burgel · 2022 · Seminars in Respiratory and Critical Care Medicine
  9. Improved glucose tolerance after initiation of Elexacaftor / Tezacaftor / Ivacaftor in adults with cystic fibrosis
    Carolin Steinack, Matthias Ernst, Felix Beuschlein, René Hage, Maurice Roeder, Macé Matthew Schuurmans, Christoph Schmid, Thomas Gaisl · 2023 · Journal of Cystic Fibrosis
  10. Reduction in abdominal symptoms (CFAbd-Score), faecal M2-pyruvate-kinase and Calprotectin over one year of treatment with Elexacaftor-Tezacaftor-Ivacaftor in people with CF aged ≥12 years – The RECOVER study
    Jochen Georg Mainz, Karen Lester, Basil Elnazir, Michael Williamson, Edward F. McKone, D. Cox, Barry M. Linnane, Carlos Zagoya, Franziska Duckstein, Anton Barucha et al. · 2023 · Journal of Cystic Fibrosis
  11. Impact of elexacaftor/tezacaftor/ivacaftor therapy on lung clearance index and magnetic resonance imaging in children with cystic fibrosis and one or twoF508delalleles
    Mirjam Stahl, Martha Dohna, Simon Y. Graeber, Olaf Sommerburg, Diane Miriam Renz, Sophia Theres Pallenberg, Andreas Voskrebenzev, Katharina Schütz, Gesine Hansen, Felix Doellinger et al. · 2024 · European Respiratory Journal
  12. The human respiratory tract microbial community structures in healthy and cystic fibrosis infants
    Marie‐Madlen Pust, Lutz Wiehlmann, Colin Davenport, Isa Rudolf, Anna‐Maria Dittrich, Burkhard Tümmler · 2020 · npj Biofilms and Microbiomes
  13. Pharmacological Modulation of Ion Channels for the Treatment of Cystic Fibrosis
    Madalena C. Pinto, Iris A. L. Silva, Miriam Frankenthal Figueira, Margarida Duarte Amaral, Miquéias Lopes‐Pacheco · 2021 · Journal of Experimental Pharmacology
  14. Fertility, Pregnancy and Lactation Considerations for Women with CF in the CFTR Modulator Era
    Raksha Jain, Jennifer L. Taylor‐Cousar · 2021 · Journal of Personalized Medicine
  15. Realizing the Dream of Molecularly Targeted Therapies for Cystic Fibrosis
    Francis S. Collins · 2019 · New England Journal of Medicine
  16. Small-molecule drugs for cystic fibrosis: Where are we now?
    Onofrio Laselva, Lorenzo Guerra, Stefano Castellani, Maria Favia, Sante Di Gioia, Massimo Conese · 2021 · Pulmonary Pharmacology & Therapeutics
  17. Cystic fibrosis transmembrane conductance regulator modulators and the exocrine pancreas: A scoping review
    Mitchell L. Ramsey, Susan S. Li, Luis F. Lara, Yevgeniya Gokun, Venkata Sandeep Akshintala, Darwin L. Conwell, John Heintz, Stephen E. Kirkby, Karen McCoy, Georgios I. Papachristou et al. · 2022 · Journal of Cystic Fibrosis
  18. Individualized approach to elexacaftor/tezacaftor/ivacaftor dosing in cystic fibrosis, in response to self-reported anxiety and neurocognitive adverse events: A case series
    Hisham Ibrahim, Hammad Danish, David J. Morrissey, Kevin Frederick Deasy, M. J. H. McCarthy, James Dorgan, C. Fleming, Ciara Howlett, Sarah Twohig, Tamara Vagg et al. · 2023 · Frontiers in Pharmacology
  19. Longitudinal Effects of Elexacaftor/Tezacaftor/Ivacaftor
    Sonia Graziano, Francesca Boldrini, Gaia Romana Pellicano, Francesco Milo, Fabio Majo, Luca Cristiani, Enza Montemitro, Federico Alghisi, Sergio Bella, Renato Cutrera et al. · 2023 · CHEST Journal
  20. PREFUL MRI for Monitoring Perfusion and Ventilation Changes after Elexacaftor-Tezacaftor-Ivacaftor Therapy for Cystic Fibrosis: A Feasibility Study
    Martha Dohna, Andreas Voskrebenzev, Filip Klimeš, Till F. Kaireit, Julian Glandorf, Sophia Theres Pallenberg, Felix C. Ringshausen, Gesine Hansen, Diane Miriam Renz, Frank K. Wacker et al. · 2024 · Radiology Cardiothoracic Imaging
  21. Structure-based discovery of CFTR potentiators and inhibitors
    Fangyu Liu, Anat Levit Kaplan, Jesper Levring, Jürgen Einsiedel, Stephanie Tiedt, Katharina Distler, Natalie S. Omattage, Ivan S. Kondratov, Yurii S. Moroz, Harlan Linver Pietz et al. · 2024 · Cell
  22. Effects of CFTR modulators on serum biomarkers of liver fibrosis in children with cystic fibrosis
    Steven Levitte, Yonathan Fuchs, Russell T. Wise, Zachary M. Sellers · 2023 · Hepatology Communications
  23. Precision Medicine and Its Imprecise History
    Christopher J. Phillips · 2020 · Harvard Data Science Review
  24. Doenças raras na agenda da inovação em saúde: avanços e desafios na fibrose cística
    Marise Basso Amaral, Sérgio Rêgo · 2020 · Cadernos de Saúde Pública
  25. Reduction of pulmonary exacerbations in young children with cystic fibrosis during the COVID‐19 pandemic
    Patel, Shreya, Thompson, Misty D., Slaven, James E., Sanders, Don B., Ren, Clement L. · 2021 · Pediatric Pulmonology
  26. The bidirectional relationship between CFTR and lipids
    Kirsten A. Cottrill, Carlos Miguel Farinha, Nael A. McCarty · 2020 · Communications Biology
  27. CFTR trafficking mutations disrupt cotranslational protein folding by targeting biosynthetic intermediates
    Hideki Shishido, Jae Seok Yoon, Zhongying Yang, William R. Skach · 2020 · Nature Communications
  28. Olfactory dysfunction in cystic fibrosis: Impact of CFTR modulator therapy
    Daniel M. Beswick, Stephen M. Humphries, Connor D. Balkissoon, Matthew J. Strand, Eszter K. Vladar, Vijay R. Ramakrishnan, Jennifer L. Taylor‐Cousar · 2021 · Journal of Cystic Fibrosis
  29. A survey: Understanding the health and perspectives of people with CF not benefiting from CFTR modulators
    Emily Kramer‐Golinkoff, Amanda Camacho, Liza Kramer, Jennifer L. Taylor‐Cousar · 2022 · Pediatric Pulmonology
  30. Real-life impact of highly effective CFTR modulator therapy in children with cystic fibrosis
    Margarete Olivier, Alexandra Kavvalou, Matthias Welsner, Raphael Hirtz, Svenja Straßburg, Sivagurunathan Sutharsan, Florian Stehling, Mathis Steindor · 2023 · Frontiers in Pharmacology
  31. Regulation of CFTR Biogenesis by the Proteostatic Network and Pharmacological Modulators
    Samuel K. Estabrooks, Jeffrey L. Brodsky · 2020 · International Journal of Molecular Sciences
  32. Physiologically Based Pharmacokinetic Modeling of CFTR Modulation in People with Cystic Fibrosis Transitioning from Mono or Dual Regimens to Triple-Combination Elexacaftor/Tezacaftor/Ivacaftor
    Alice Tsai, Shu-Pei Wu, Eric L. Haseltine, Sanjeev Kumar, Samuel M. Moskowitz, Paul Panorchan, Kushal Shah · 2020 · Pulmonary Therapy
  33. A current review of the safety of cystic fibrosis transmembrane conductance regulator modulators
    Elizabeth Marie Gavioli, Nerli Guardado, Farah Haniff, Nouran Deiab, Etty Vider · 2020 · Journal of Clinical Pharmacy and Therapeutics
  34. Potential of the Electronic Nose for the Detection of Respiratory Diseases with and without Infection
    Johann-Christoph Licht, Hartmut Grasemann · 2020 · International Journal of Molecular Sciences
  35. Airway Epithelial Inflammation In Vitro Augments the Rescue of Mutant CFTR by Current CFTR Modulator Therapies
    Martina Gentzsch, Deborah M. Cholon, Nancy L. Quinney, Mary E.B. Martino, John T. Minges, Susan E. Boyles, Tara N. Guhr Lee, Charles R. Esther, Carla M. P. Ribeiro · 2021 · Frontiers in Pharmacology
  36. Correction of Airway Stem Cells: Genome Editing Approaches for the Treatment of Cystic Fibrosis
    Nicholas E. King, Shingo Suzuki, C. Barillà, Finn J Hawkins, Scott H. Randell, Susan D. Reynolds, Barry R. Stripp, Brian R. Davis · 2020 · Human Gene Therapy
  37. Biological insights in non-small cell lung cancer
    Rafael Rosell, Anisha S Jain, Jordi Codony‐Servat, Eloisa Jantus Lewintre, Blake Morrison, Jordi Barretina, María González‐Cao · 2023 · Cancer Biology and Medicine
  38. Body mass index and nutritional intake following Elexacaftor/Tezacaftor/Ivacaftor modulator therapy in adults with cystic fibrosis
    L.R. Caley, Heledd Jarosz-Griffiths, L. Smith, L. Gale, Joanne Barrett, Laura Kinsey, V. Davey, M. Nash, Andrew M. Jones, Joanna L. Whitehouse et al. · 2023 · Journal of Cystic Fibrosis
  39. CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare CFTR Genotypes
    Juliet W. Lefferts, Marlou C. Bierlaagh, Suzanne Kroes, Natascha D.A. Nieuwenhuijze, Heleen N. Sonneveld van Kooten, Paul J Niemöller, Tibo F. Verburg, Hettie M. Janssens, Danya Muilwijk, Sam F.B. van Beuningen et al. · 2023 · International Journal of Molecular Sciences
  40. Benchmarking AlphaMissense pathogenicity predictions against cystic fibrosis variants
    McDonald, Eli Fritz, Oliver, Kathryn E., Schlebach, Jonathan P., Meiler, Jens, Plate, Lars · 2024 · PLOS ONE
  41. Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome Editing
    Steven Erwood, Onofrio Laselva, Teija M.I. Bily, Reid A. Brewer, Alexandra H. Rutherford, Christine E. Bear, Evgueni A. Ivakine · 2020 · Molecular Therapy — Methods & Clinical Development
  42. Biliary disease and cholecystectomy after initiation of elexacaftor/ivacaftor/tezacaftor in adults with cystic fibrosis
    Julie Safirstein, Jonathan J. Grant, Emily Siu Clausen, Deepika Savant, Rebecca Dezube, Gina Hong · 2020 · Journal of Cystic Fibrosis
  43. Challenges in the use of highly effective modulator treatment for cystic fibrosis
    Kathleen J. Ramos, Joseph M. Pilewski, Jennifer L. Taylor‐Cousar · 2021 · Journal of Cystic Fibrosis
  44. DNAJB12 and Hsp70 triage arrested intermediates of N1303K-CFTR for endoplasmic reticulum-associated autophagy
    Lihua He, Andrew S. Kennedy, Scott A. Houck, Andrei A. Aleksandrov, Nancy L. Quinney, Alexandra Cyr-Scully, Deborah M. Cholon, Martina Gentzsch, Scott H. Randell, Hong Yu Ren et al. · 2021 · Molecular Biology of the Cell
  45. Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype
    Vito Terlizzi, Felice Amato, Chiara Castellani, Béatrice Ferrari, Luis J. V. Galietta, Giuseppe Castaldo, Giovanni Taccetti · 2021 · Molecular Genetics & Genomic Medicine
  46. Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review
    Jamie M Duckers, Beth A. Lesher, Teja Thorat, Eleanor J. Lucas, Lisa J. McGarry, Keval Chandarana, Fosca De Iorio · 2021 · Journal of Clinical Medicine
  47. Revisiting Host-Pathogen Interactions in Cystic Fibrosis Lungs in the Era of CFTR Modulators
    Carla M. P. Ribeiro, Matthew G. Higgs, Marianne S. Muhlebach, Matthew C. Wolfgang, Monica Borgatti, Ilaria Lampronti, Giulio Cabrini · 2023 · International Journal of Molecular Sciences
  48. Bacteriophage Therapy for Pan-Drug-Resistant Pseudomonas aeruginosa in Two Persons With Cystic Fibrosis
    Andrea Hahn, Iman R. Sami, Hollis R. Chaney, Anastassios C. Koumbourlis, Coralee Del Valle Mojica, Claire I. Cochrane, Benjamin K. Chan, Jonathan L. Koff · 2023 · Journal of Investigative Medicine High Impact Case Reports
  49. Pharmacological approaches for targeting cystic fibrosis nonsense mutations
    Jyoti Sharma, Kim M. Keeling, Steven Mark Rowe · 2020 · European Journal of Medicinal Chemistry
  50. CRISPR/Cas9 gene editing therapies for cystic fibrosis
    Carina Graham, Stephen L. Hart · 2021 · Expert Opinion on Biological Therapy
  51. Chest computed tomography improvement in patients with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor: Early report
    Romain Bec, Martine Reynaud‐Gaubert, François Arnaud, Romain Naud, Nadine Desmazes Dufeu, Mathieu Di Biscéglie, Benjamin Coiffard, Jean‐Yves Gaubert, Julien Bermudez, Paul Habert · 2022 · European Journal of Radiology
  52. Impact of elexacaftor/tezacaftor/ivacaftor on bacterial cultures from people with cystic fibrosis
    Michael R. Beck, Douglas B. Hornick, Tahuanty A. Pena, Sachinkumar B. Singh, Brittany A. Wright · 2023 · Pediatric Pulmonology
  53. Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rare CFTR variants: a viewpoint
    Pierre‐Régis Burgel, Isabelle Sermet‐Gaudelus, Emmanuelle Girodon, R. Kanaan, Jean Le Bihan, Natascha Remus, Bruno Ravoninjatovo, Dominique Grenet, Michele Porzio, Véronique Houdouin et al. · 2024 · European Respiratory Journal
  54. Cystic fibrosis and primary ciliary dyskinesia: Similarities and differences
    Rute Pereira, Telma Barbosa, Ana Lúcia Cardoso, Rosália Sá, Mário Sousa · 2023 · Respiratory Medicine
  55. Personalized CFTR Modulator Therapy for G85E and N1303K Homozygous Patients with Cystic Fibrosis
    Simon Y. Graeber, Anita Balázs, Niklas Ziegahn, Tihomir Rubil, Constanze Vitzthum, Linus Piehler, Marika Drescher, Kathrin Seidel, Alexander Rohrbach, Jobst Fridolin Roehmel et al. · 2023 · International Journal of Molecular Sciences
  56. Evaluation of elexacaftor–tezacaftor–ivacaftor treatment in individuals with cystic fibrosis and CFTRN1303K in the USA: a prospective, multicentre, open-label, single-arm trial
    George Marty Solomon, Rachel W. Linnemann, Rachel Rich, Ashleigh Streby, B. Buehler, Eric Hunter, Kadambari Vijaykumar, William R. Hunt, John J. Brewington, András Rab et al. · 2024 · The Lancet Respiratory Medicine
  57. Comparison of Organoid Swelling and In Vivo Biomarkers of CFTR Function to Determine Effects of Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for the F508del Mutation
    Simon Y. Graeber, Peter van Mourik, Annelotte M. Vonk, Evelien Kruisselbrink, Stephanie Hirtz, Cornelis Korstiaan van der Ent, Marcus Alexander Mall, Jeffrey Matthijn Beekman · 2020 · American Journal of Respiratory and Critical Care Medicine
  58. CFTR Modulator Therapy with Lumacaftor/Ivacaftor Alters Plasma Concentrations of Lipid-Soluble Vitamins A and E in Patients with Cystic Fibrosis
    Olaf Sommerburg, Susanne Hämmerling, S. Philipp Schneider, Jürgen G. Okun, Claus‐Dieter Langhans, Patricia Leutz-Schmidt, Mark Oliver Wielpütz, Werner Siems, Simon Y. Gräber, Marcus Alexander Mall et al. · 2021 · Antioxidants
  59. A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants
    Guido Veit, Tony Velkov, Haijin Xu, Nathalie Vadeboncoeur, Lara Bilodeau, Elias Matouk, Gergely L. Lukács · 2021 · Journal of Personalized Medicine
  60. Family‐building and parenting considerations for people with cystic fibrosis
    Traci M. Kazmerski, Natalie E. West, Raksha Jain, Ahmet Uluer, Anna M. Georgiopoulos, Moira L. Aitken, Jennifer L. Taylor‐Cousar · 2021 · Pediatric Pulmonology
  61. Cancer in Cystic Fibrosis: A Narrative Review of Prevalence, Risk Factors, Screening, and Treatment Challenges
    Patrick Maisonneuve, Albert B. Lowenfels · 2021 · CHEST Journal
  62. CFTR and Gastrointestinal Cancers: An Update
    Rahul Bhattacharya, Zachary Blankenheim, Patricia M. Scott, Robert T. Cormier · 2022 · Journal of Personalized Medicine
  63. Weight a minute: Exploring the effect on weight and body composition after the initiation of elexacaftor/tezacaftor/ivacaftor in adults with CF
    David Proud, Jamie M Duckers · 2023 · Journal of Cystic Fibrosis
  64. Cystic Fibrosis Patients with F508del/Minimal Function Genotype: Laboratory and Nutritional Evaluations after One Year of Elexacaftor/Tezacaftor/Ivacaftor Treatment
    Vincenzo Carnovale, Filippo Scialò, Monica Gelzo, Paola Iacotucci, Felice Amato, Federica Zarrilli, Assunta Celardo, Giuseppe Castaldo, Gaetano Corso · 2022 · Journal of Clinical Medicine
  65. Antisense Oligonucleotide Therapeutics for Cystic Fibrosis: Recent Developments and Perspectives
    Young Jin Kim, Adrian R. Krainer · 2023 · Molecules and Cells
  66. Drug-induced liver injury associated with elexacaftor/tezacaftor/ivacaftor: A pharmacovigilance analysis of the FDA adverse event reporting system (FAERS)
    Alan Shi, Harold Nguyen, Chiaoyun Benson Kuo, Paul M. Beringer · 2024 · Journal of Cystic Fibrosis
  67. Functional Consequences of CFTR Interactions in Cystic Fibrosis
    Yashaswini Ramananda, Anjaparavanda P. Naren, Kavisha Arora · 2024 · International Journal of Molecular Sciences
  68. The Precision Medicine Era of Bronchiectasis
    Sanjay Haresh Chotirmall, James Duncan Chalmers · 2024 · American Journal of Respiratory and Critical Care Medicine
  69. The preclinical discovery and development of the combination of ivacaftor + tezacaftor used to treat cystic fibrosis
    Lorenzo Guerra, Maria Favia, Sante Di Gioia, Onofrio Laselva, Arianna Bisogno, Valeria Casavola, Carla Colombo, Massimo Conese · 2020 · Expert Opinion on Drug Discovery
  70. Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations
    Munck, Anne, Kerem, Eitan, Ellemunter, Helmut, Campbell, Daniel, Wang, Linda T., Ahluwalia, Neil, Owen, Caroline A., Wainwright, Claire · 2020 · Journal of Cystic Fibrosis
  71. Targeting the Heme Oxygenase 1/Carbon Monoxide Pathway to Resolve Lung Hyper-Inflammation and Restore a Regulated Immune Response in Cystic Fibrosis
    Caterina Di Pietro, Hasan Halit Öz, Thomas S. Murray, Emanuela M. Bruscia · 2020 · Frontiers in Pharmacology
  72. CFTR Modulators: Does One Dose Fit All?
    Renske van der Meer, Erik B. Wilms, Harry G.M. Heijerman · 2021 · Journal of Personalized Medicine
  73. A new platform for high-throughput therapy testing on iPSC-derived lung progenitor cells from cystic fibrosis patients
    Jia Xin Jiang, Leigh Wellhauser, Onofrio Laselva, Irina Utkina, Zoltán Bozóky, Tarini Nawamalie Abeysinghe Gunawardena, Zoe Ngan, Sunny Xia, Michelle Di Paola, Paul D. W. Eckford et al. · 2021 · Stem Cell Reports
  74. Physiologically‐Based Pharmacokinetic‐Led Guidance for Patients With Cystic Fibrosis Taking Elexacaftor‐Tezacaftor‐Ivacaftor With Nirmatrelvir‐Ritonavir for the Treatment of COVID‐19
    Eunjin Hong, Lisa M. Almond, Peter S. Chung, Adupa P. Rao, Paul M. Beringer · 2022 · Clinical Pharmacology & Therapeutics
  75. TMEM16A (ANO1) as a therapeutic target in cystic fibrosis
    Luis J. V. Galietta · 2022 · Current Opinion in Pharmacology
  76. Small-molecule eRF3a degraders rescue CFTR nonsense mutations by promoting premature termination codon readthrough
    Rhianna E. Lee, Catherine A. Lewis, Lihua He, Emily C. Bulik‐Sullivan, Samuel C. Gallant, Teresa Mascenik, Hong Dang, Deborah M. Cholon, Martina Gentzsch, Lisa C. Morton et al. · 2022 · Journal of Clinical Investigation
  77. Nutritional Status and Circulating Levels of Fat-Soluble Vitamins in Cystic Fibrosis Patients: A Cohort Study and Evaluation of the Effect of CFTR Modulators
    Michela Francalanci, Vito Terlizzi, Cristina Fevola, G. Di Rosa, Valentina Pierattini, Elena Roselli, Paolo Bonomi, Maria Chiara Cavicchi, Valeria Galici, Anna Silvia Neri et al. · 2023 · Children
  78. Cystic Fibrosis-Related Diabetes Workshop: research priorities spanning disease pathophysiology, diagnosis, and outcomes
    Putman, Melissa S., Norris, Andrew W., Hull, Rebecca L., Rickels, Michael R., Sussel, Lori, Blackman, Scott M., Chan, Christine L., Ode, Katie Larson, Daley, Tanicia, Stecenko, Arlene A. et al. · 2023 · Diabetes Care
  79. Impact of Elexacaftor–Tezacaftor–Ivacaftor on lung disease in cystic fibrosis
    Courtney A. Gushue, Mariah Eisner, Shasha Bai, Terri Johnson, Melissa Holtzlander, Karen McCoy, Shahid Ijaz Sheikh · 2023 · Pediatric Pulmonology
  80. Heterogeneity in response to Elexacaftor/Tezacaftor/Ivacaftor in people with cystic fibrosis
    Gianfranco Alicandro, Andrea Gramegna, Federica Bellino, Calogero Sathya Sciarrabba, Chiara Lanfranchi, Martina Contarini, Mariangela Retucci, Valeria Daccò, Francesco Blasi · 2024 · Journal of Cystic Fibrosis
  81. Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function
    Pierre‐Régis Burgel, I. Durieu, R. Chiron, Laurent Mély, Anne Prévötat, M. Murris‐Espin, Michele Porzio, Michel Abély, Philippe Reix, Christophe Marguet et al. · 2020 · Journal of Cystic Fibrosis
  82. Early Diagnosis and Intervention in Cystic Fibrosis: Imagining the Unimaginable
    Andrea M. Coverstone, Thomas W. Ferkol · 2021 · Frontiers in Pediatrics
  83. Dysfunctional Inflammation in Cystic Fibrosis Airways: From Mechanisms to Novel Therapeutic Approaches
    Alessandra Ghigo, Giulia Prono, Elisa Riccardi, Virginia De Rose · 2021 · International Journal of Molecular Sciences
  84. Treatment of Pulmonary Disease of Cystic Fibrosis: A Comprehensive Review
    Rosa María Girón Moreno, Marta María García Clemente, Layla Diab-Cáceres, Adrián Martínez-Vergara, Miguel Ángel Martínez‐García, Rosa Mar Gómez-Punter · 2021 · Antibiotics
  85. Short-term effect of elexacaftor-tezacaftor-ivacaftor on lung function and transplant planning in cystic fibrosis patients with advanced lung disease
    B Bermingham, A. Rueschhoff, Gregory A. Ratti, Andrew NeSmith, Dana L. Goodwin, Shelly L. Gray, Patrick A. Flume, George Marty Solomon, Cohen L, Bryan A. Garcia · 2021 · Journal of Cystic Fibrosis
  86. CFTR modulator use in post lung transplant recipients
    Lauryn A. Benninger, Cesar Trillo‐Alvarez, Jorge E. Lascano · 2021 · The Journal of Heart and Lung Transplantation
  87. Onset of systemic arterial hypertension after initiation of elexacaftor/tezacaftor/ivacaftor in adults with cystic fibrosis: A case series
    Andrea Gramegna, Claudia De Petro, Gloria Leonardi, Martina Contarini, Francesco Amati, Roberto Meazza, Stefano Carugo, Francesco Blasi · 2022 · Journal of Cystic Fibrosis
  88. Advances in Preclinical In Vitro Models for the Translation of Precision Medicine for Cystic Fibrosis
    Iris A. L. Silva, Onofrio Laselva, Miquéias Lopes‐Pacheco · 2022 · Journal of Personalized Medicine
  89. Patient-derived cell models for personalized medicine approaches in cystic fibrosis
    Anabela Santo Ramalho, Felice Amato, Martina Gentzsch · 2022 · Journal of Cystic Fibrosis
  90. Moving the Dial on Airway Inflammation in Response to Trikafta in Adolescents with Cystic Fibrosis
    Agathe Lepissier, Anne Sophie Bonnel, Nathalie Wizla, Laurence M. Weiss, Marie Mittaine, Katia Bessaci, Eitan Kerem, Véronique Houdouin, Philippe Reix, Christophe Marguet et al. · 2023 · American Journal of Respiratory and Critical Care Medicine
  91. The role of modulators in cystic fibrosis related diabetes
    Lina Merjaneh, Sana Hasan, Nader R. Kasim, Katie Larson Ode · 2021 · Journal of Clinical & Translational Endocrinology
  92. Elexacaftor – Tezacaftor – Ivacaftor treatment improves systemic infection parameters and Pseudomonas aeruginosa colonization rate in patients with cystic fibrosis a monocentric observational study
    Alexander Schnell, Hannah Hober, Natalie Kaiser, Renate Ruppel, Annika Geppert, Christina Tremel, Julia Sobel, Erika Plattner, Joachim Woelfle, André Hoerning · 2023 · Heliyon
  93. Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective
    Nicole Mayer-Hamblett, John Paul Clancy, Raksha Jain, Scott H. Donaldson, Isabelle Fajac, Christopher Hooper Goss, Deepika Polineni, Felix A. Ratjen, Bradley S. Quon, Edith T. Zemanick et al. · 2023 · The Lancet Respiratory Medicine
  94. Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care
    Marian L. Henderson, Jacob K. Zieba, Xiaopeng Li, Daniel B. Campbell, Michael R. Williams, Daniel L. Vogt, Caleb P. Bupp, Yvonne M. Edgerly, Surender Rajasekaran, Nicholas Hartog et al. · 2024 · BioTech
  95. Update on advances in cystic fibrosis towards a cure and implications for primary care clinicians
    Vito Terlizzi, Philip M. Farrell · 2024 · Current problems in pediatric and adolescent health care
  96. Cystic fibrosis: new challenges and perspectives beyond elexacaftor/tezacaftor/ivacaftor
    Vito Terlizzi, Miquéias Lopes‐Pacheco · 2025 · Therapeutic Advances in Respiratory Disease
  97. Phenotyping of Rare CFTR Mutations Reveals Distinct Trafficking and Functional Defects
    Marjolein M. Ensinck, Liesbeth De Keersmaecker, Lise Heylen, Anabela Santo Ramalho, Rik Gijsbers, Ricard Farré, K. De Boeck, Frauke Christ, Zeger Debyser, Marianne Sylvia Carlon · 2020 · Cells
  98. Cystic fibrosis transmembrane conductance receptor modulator therapy in cystic fibrosis, an update
    Marie E. Egan · 2020 · Current Opinion in Pediatrics
  99. Multiple Reaction Monitoring Mass Spectrometry for the Drug Monitoring of Ivacaftor, Tezacaftor, and Elexacaftor Treatment Response in Cystic Fibrosis: A High-Throughput Method
    Felisa Reyes‐Ortega, Fiona Qiu, Elena K. Schneider‐Futschik · 2020 · ACS Pharmacology & Translational Science
  100. The era of CFTR modulators: improvements made and remaining challenges
    Sara Cuevas Ocaña, Onofrio Laselva, Julie Avolio, Raffaella Nenna · 2020 · Breathe