Results 1 to 10 of about 4,751 (165)

Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor [PDF]

Frontiers in Pharmacology, 2023
Triple combination therapy with the CFTR modulators elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) has been qualified as a game changer in cystic fibrosis (CF).
Burkhard Tümmler, Burkhard Tümmler
doaj   +3 more sources

Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials [PDF]

Lancet Respiratory Medicine,the, 2023
BACKGROUND: Elexacaftor-tezacaftor-ivacaftor has been shown to be safe and efficacious in people with cystic fibrosis and at least one F508del allele. Our aim was to identify a novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator ...
et al.,, Rubenstein, Ronald C, Uluer, Ahmet Z   +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

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

People with cystic fibrosis with high sputum neutrophil elastase on elexacaftor-tezacaftor-ivacaftor exhibit worse pulmonary function and pro-inflammatory airway milieu [PDF]

Scientific Reports
Cystic fibrosis (CF) is a genetic condition affecting over 100,000 individuals worldwide. Lung disease is the main cause of mortality in CF, with chronic neutrophilic inflammation as a hallmark.
Alexandre Cammarata-Mouchtouris, Diego Moncada Giraldo, Genoah L. Collins, Maria M. Parrilla, Susan O. Kim, Sarah Mansour, Vincent D. Giacalone, Kirsten A. Cottrill, Rabindra Tirouvanziam, Joshua D. Chandler   +9 more
doaj   +2 more sources

Real-world population pharmacokinetics of tezacaftor-ivacaftor in children with cystic fibrosis: The SYM-CF study. [PDF]

Br J Clin Pharmacol
Aims: The clinical effectiveness of tezacaftor-ivacaftor in children with cystic fibrosis (cwCF) varies; some patients respond while others do not or have adverse effects.
Vonk SEM, Terheggen-Lagro SWJ, Haarman EG, Janssens HM, Maitland-van der Zee AH, Kemper EM, Mathôt RAA, Amsterdam Mucociliary Clearance Disease (AMCD) research group.   +7 more
europepmc   +3 more sources

Vanzacaftor–tezacaftor–deutivacaftor for children aged 6–11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial [PDF]

Lancet Respiratory Medicine,the
BACKGROUND: In phase 2 trials in people with cystic fibrosis aged 18 years and older, vanzacaftor–tezacaftor–deutivacaftor has been shown to be a safe and effective, once-daily cystic fibrosis transmembrane conductance regulator (CFTR) modulator ...
Chen, YC, Davies, G, Hoppe, JE, Jensen, R, Kasi, AS, Mall, MA, McKone, EF, Pittman, JE, Ramsey, B, Robinson, P, Rodriguez-Romero, V, Salinas, DB, Sosnay, PR, Taylor-Cousar, JL, Thia, LP, Tirakitsoontorn, P, Tullis, E, Zhu, J   +17 more
exaly   +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