A Review of Trikafta: Triple Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy
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TLDR
Trikafta as discussed by the authors is the third FDA-approved drug that targets the F508del mutation of the CFTR gene, which increases the activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and reduces the mortality and morbidity rates in CF patients.Abstract:
Cystic fibrosis (CF) is a potentially fatal genetic disease that causes serious lung damage. With time, researchers have a more complete understanding of the molecular-biological defects that underlie CF. This knowledge is leading to alternative approaches regarding the treatment of this condition. Trikafta is the third FDA-approved drug that targets the F508del mutation of the CFTR gene. The drug is a combination of three individual drugs which are elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA). This trio increases the activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and reduces the mortality and morbidity rates in CF patients. The effectiveness of Trikafta, seen in clinical trials, outperforms currently available therapies in terms of lung function, quality of life, sweat chloride reduction, and pulmonary exacerbation reduction. The safety and efficacy of CFTR modulators in children with CF have also been studied. Continued evaluation of patient data is needed to confirm its long-term safety and efficacy. In this study, we will focus on reviewing data from clinical trials regarding the benefits of CFTR modulator therapy. We address the impact of Trikafta on lung function, pulmonary exacerbations, and quality of life. Adverse events of the different CFTR modulators are discussed.read more
Citations
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Journal ArticleDOI
Molecular Structures Reveal Synergistic Rescue of Δ508 CFTR by Trikafta Modulators
Karol Fiedorczuk,Jue Chen +1 more
TL;DR: Cryo-electron microscopy structures of Δ508 CFTR in the absence and presence of CFTR modulators are presented to illustrate how the different modulators in Trikafta synergistically rescue Δ508CFTR structure and function.
Journal ArticleDOI
Impact of CFTR Modulators on Beta-Cell Function in Children and Young Adults with Cystic Fibrosis
Claudia Piona,Enza Mozzillo,Antonella Tosco,Sonia Volpi,Francesco Maria Rosanio,Chiara Cimbalo,Adriana Franzese,Valeria Raia,Chiara Zusi,Federica Emiliani,Maria Linda Boselli,Maddalena Trombetta,Riccardo C. Bonadonna,Marco Cipolli,Claudio Maffeis +14 more
TL;DR: Treatment of CF patients with CFTR modulators does not significantly ameliorate glucose homeostasis and/or any of its direct determinants, and glucose regulation was stable in both groups.
Journal ArticleDOI
Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study
G Migliorisi,Mirella Collura,Francesca Ficili,Tiziana Pensabene,Dafne Bongiorno,A. Collura,F. Di Bernardo,Stefania Stefani +7 more
TL;DR: A one-year case-control study that involved 26 patients with at least one F508del mutation and aimed to define the clinical and especially microbiological implications of treatment administration, which provided significant clinical benefits in terms of respiratory, pancreatic, and sweat function.
Journal ArticleDOI
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,A. Celardo,Giuseppe Castaldo,Gaetano Corso +8 more
TL;DR: In this paper , the effect of triple combination elexacaftor/tezacaftors/ivacaftors on liver function and nutrient absorption with a focus on lipid metabolism was investigated.
Journal ArticleDOI
Chronic cough in cystic fibrosis: the effect of modulator therapy on objective 24-h cough monitoring
TL;DR: An early and dramatic improvement in the objective measure of 24-h cough monitoring was observed, providing noninvasive, objective evidence of efficacy and compliance in CF.
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