Results 1 to 10 of about 2,795 (144)

Physiologically Based Pharmacokinetic Modeling of Elexacaftor/Tezacaftor/Ivacaftor in Infants With Cystic Fibrosis [PDF]

CPT: Pharmacometrics & Systems Pharmacology
Ivacaftor is the only cystic fibrosis transmembrane conductance regulator modulator approved for infants ≥ 1 month. The elexacaftor/tezacaftor/ivacaftor combination, approved for children aged ≥ 2 years, has been shown to significantly slow CF ...
Ngoc Hoa Truong, Sihem Benaboud, Naïm Bouazza, Eric Deneuville, Anne Mornand, Philippe Reix, Mélanie Ribault, Pierre‐Régis Burgel, Léo Froelicher‐Bournaud, Steeve Rouillon, Mohammed Rohi Sanoufi, Gabrielle Lui, Saïk Urien, Jean‐Marc Tréluyer, Isabelle Sermet‐Gaudelus, Frantz Foissac, Modul‐CF study group   +16 more
doaj   +2 more sources

Simultaneous Quantification of Ivacaftor, Tezacaftor, and Elexacaftor in Cystic Fibrosis Patients’ Plasma by a Novel LC–MS/MS Method

Biomedicines, 2023
The new breakthrough cystic fibrosis (CF) drug combination of ivacaftor (IVA), tezacaftor (TEZ), and elexacaftor (ELX), namely “caftor” drugs, directly modulates the activity and trafficking of the defective CF transmembrane conductance regulator protein
Francesca Mattioli, Sebastiano Barco, Federico Cresta   +2 more
exaly   +3 more sources

Comparative efficacy and safety of CFTR modulators for people with cystic fibrosis with phe508del mutation: a systematic review and bayesian network meta-analysisResearch in context [PDF]

EClinicalMedicine
Summary: Background: The development of cystic fibrosis transmembrane conductance regulator (CFTR) modulators (correctors and potentiators) emerged as a promising approach, aiming to restore CFTR protein function. A lack of head-to-head trials comparing
Mohammed Safeer V S, Simran Behl, Pankaj C. Vaidya, Pawan Tiwari, Saroj Kundan Bharti, Najiya Nahan, Jitendra Kumar Sahu, Dipika Bansal   +7 more
doaj   +2 more sources

Exposure to CFTR Modulators During Pregnancy in Cystic Fibrosis: Four Cases to Highlight Neonatal Diagnostic Challenges and Outcomes [PDF]

International Journal of Neonatal Screening
CFTR modulators have transformed the clinical evolution of patients with CF. The number of pregnancies is increasing in women with CF, most of whom are now treated with CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ETI) or Tezacaftor ...
Louis Domenach, Adrien Pagin, Camille Cisterne, Marie-Pierre Audrezet, Laure Couderc, Laetitia Monteil, Léa Roditis, Marlène Murris, Julie Macey, Michael Fayon, Stéphanie Bui, Marie-Pierre Reboul   +11 more
doaj   +2 more sources

Long-term impact of Elexacaftor/Tezacaftor/ivacaftor on pulmonary, nutritional and metabolic outcomes in homozygous F508del cystic fibrosis patients: A real-world cohort study. [PDF]

Br J Clin Pharmacol
Aim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Perrotta N, Fiorito LA, Vescovo R, Virgilio A, Amato G, Cimino G.   +5 more
europepmc   +2 more sources

Long-Term Adherence to Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis [PDF]

Pulmonary Therapy
Introduction Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators are the cornerstone treatment for CF, but nonadherence can reduce their effect.
Carina M. E. Hansen, Badr Zendaky, Annelies M. Zwitserloot, Patricia M. L. A. van den Bemt, Job F. M. van Boven   +4 more
doaj   +2 more sources

Large variations in total and allele-specific transcript expression in a disease mutation-independent manner [PDF]

Scientific Reports
Individuals with monogenic diseases, even those with identical disease-causing mutations, exhibit considerable clinical heterogeneity in severity and outcomes.
Moritz Freyberg, Merete Bewig, Giovana Bavia Bampi, Candela Manfredi, Disha Joshi, Robert Rauscher, Jeong S. Hong, Jörg Große-Onnebrink, Sivagurunathan Sutharsan, Florian Stehling, Ingrid Bobis, Manfred Ballmann, Eric J. Sorscher, Zoya Ignatova   +13 more
doaj   +2 more sources

Real-life impact of highly effective CFTR modulator therapy in children with cystic fibrosis

Frontiers in Pharmacology, 2023
Introduction: Recently, cystic fibrosis transmembrane regulator modulator therapy with elexacaftor/tezacaftor/ivacaftor has become available for children with cystic fibrosis (CF) carrying at least one F508del mutation.Objective: To assess the ...
Margarete Olivier, Alexandra Kavvalou, Matthias Welsner, Raphael Hirtz, Svenja Straßburg, Sivagurunathan Sutharsan, Florian Stehling, Mathis Steindor   +7 more
doaj   +1 more source

Cystic fibrosis modulator therapy can reverse cystic bronchiectasis

Respirology Case Reports, 2023
Bronchiectasis is often considered progressive and irreversible, so cases of regression or reversal are an important step in understanding the underlying pathophysiological mechanisms.
Peter G. Middleton, Nicholas J. Simmonds
doaj   +1 more source

Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

Children, 2023
Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic ...
Nikoletta Kapouni, Maria Moustaki, Konstantinos Douros, Ioanna Loukou   +3 more
doaj   +1 more source