Results 61 to 70 of about 2,795 (144)

Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor

Molecules
The monogenic rare disease Cystic Fibrosis (CF) is caused by mutations in the gene encoding the CF transmembrane conductance (CFTR) protein, an anion channel expressed at the apical plasma membrane of epithelial cells.
Filipa C. Ferreira, Camilla D. Buarque, Miquéias Lopes-Pacheco   +2 more
doaj   +1 more source

The relationship between the pharmacogenetics and pharmacokinetics of CFTR modulators in a population of children with cystic fibrosis

Фармация и фармакология (Пятигорск)
The triple combination of elexacaftor / tezacaftor / ivacaftor is one of the key tools for targeted pharmacotherapy of cystic fibrosis. In the pediatric population, its use may be accompanied by a variable therapeutic response, which may be due to age ...
S. K. Zyryanov, E. I. Kondratyeva, E. K. Zhekaite, O. I. Butranova, P. O. Bochkov   +4 more
doaj   +1 more source

Cystic Fibrosis: New Trends in Therapy Methods

Архивъ внутренней медицины
This review provides information on recent advancements in the treatment of cystic fi brosis and presents interim results from ongoing clinical trials. Various scientifi c databases, including Scopus, Web of Science, and EMBASE, were utilized during the ...
P. A. Suchkova, S. A. Panova, O. Ya. Lisenko, K. P. Raevskij   +3 more
doaj   +1 more source

Ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor–ivacaftor for treating cystic fibrosis: a systematic review and economic evaluation

Health Technology Assessment
Background Cystic fibrosis is a life-limiting genetic condition that affects over 9000 people in England. Cystic fibrosis is usually diagnosed through newborn screening and causes symptoms throughout the body, including the lungs and digestive system ...
Steven J Edwards, Benjamin G Farrar, Kate Ennis, Nicole Downes, Victoria Wakefield, Isaac Mackenzie, Archie Walters, Tracey Jhita   +7 more
doaj   +1 more source

Divergent neurobehavioral effects of CFTR modulators elexacaftor and ivacaftor in mice

Acta Psychologica
Recent advances in cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapies have markedly improved survival and quality of life for people with cystic fibrosis (CF).
Qian Ge, Amy Fagan, Hui Lu, Jianyang Du, Leah R. Reznikov   +4 more
doaj   +1 more source

The F508del-CFTR trafficking correctors elexacaftor and tezacaftor are CFTR-independent Ca2+-mobilizing agonists normalizing abnormal Ca2+ levels in human airway epithelial cells

Respiratory Research
Background Cystic fibrosis (CF) is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) channel. For people with CF (pwCF) affected by the most common pathogenic variant F508del, a tritherapy, named ...
Manuella Lévêque, Sandra Mirval, Christine Barrault, Isabelle Fixe, Christelle Coraux, Edouard Sage, Frédéric Becq, Clarisse Vandebrouck   +7 more
doaj   +1 more source

ID-44 Perfil de Distribuição da Terapia Tripla para Tratamento da Fibrose Cística no 2º Semestre de 2024

Jornal de Assistência Farmacêutica e Farmacoeconomia
Objetivo: Analisar o perfil de distribuição do medicamento que representa uma tripla terapia para fibrose cística, consistindo na associação elexacaftor, tezacaftor e  ivacaftor, possuindo duas apresentações: elexacaftor 50 mg + tezacaftor 25 mg ...
Flavia Kimura Okamoto, Marcelo Azevedo Coutinho   +1 more
doaj   +1 more source

The long-term effect of elexacaftor/tezacaftor/ivacaftor on cardiorespiratory fitness in adolescent patients with cystic fibrosis: a pilot observational study

BMC Pulmonary Medicine
Background Physical activity is a crucial demand on cystic fibrosis treatment management. The highest value of oxygen uptake (VO2peak) is an appropriate tool to evaluate the physical activity in these patients.
Nela Stastna, Lenka Hrabovska, Pavel Homolka, Lukas Homola, Michal Svoboda, Kristian Brat, Libor Fila   +6 more
doaj   +1 more source

Profile of tezacaftor/ivacaftor combination and its potential in the treatment of cystic fibrosis

Therapeutics and Clinical Risk Management, 2019
Dejene Shiferaw,* Shoaib Faruqi*Department of Respiratory Medicine, Hull University Teaching Hospitals NHS Trust, Cottingham HU16 5JQ, UK *These authors contributed equally to this workCorrespondence: Shoaib FaruqiDepartment of Respiratory Medicine ...
Shiferaw D, Faruqi S
doaj  

Impact of CFTR modulator concentrations on clinical response in cystic fibrosis. [PDF]

Eur Respir J
Chalamalla AR, Baker E, Ryan KJ, Dowell A, Natt JR, Zemanick ET, Konstan MW, Mayer-Hamblett N, Acosta EP, Guimbellot JS, CHEC-SC investigators.   +10 more
europepmc   +1 more source