Cystic Fibrosis: New Trends in Therapy Methods
Архивъ внутренней медициныThis 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 +3 more
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Divergent neurobehavioral effects of CFTR modulators elexacaftor and ivacaftor in mice
Acta PsychologicaRecent 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 +4 more
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A case of severe pulmonary exacerbation in a young patient with cystic fibrosis in the era of CFTR modulators [PDF]
The introduction of CFTR modulator drugs like Elexacaftor-Tezacaftor-Ivacaftor (ETI) has transformed the management of Cystic Fibrosis (CF), significantly improving symptoms, lung function, and quality of life, while reducing reliance on intravenous ...
Alicandro, Gianfranco +8 more
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Health Technology AssessmentBackground 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 +7 more
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Quantification of Ivacaftor, Tezacaftor, Elexacaftor, and Lumacaftor and their active metabolites in plasma using UHPLC-MS/MS:Doors open to the application of therapeutic drug monitoring in cystic fibrosis treatment [PDF]
An ultra-high performance liquid chromatography-tandem mass spectrometry method was developed to quantify the cystic fibrosis transmembrane conductance regulator (CFTR) modulators ivacaftor, tezacaftor, elexacaftor, and lumacaftor and their active ...
Akkerman, Onno W +5 more
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Is Triple Combination CFTR Modulator Therapy Effective in Improving the Quality of Life of Patients with Cystic Fibrosis Who Have One Phe508del Allele? [PDF]
, 2020 Objective: The objective of this selective EBM review is to determine whether or not “Is triple combination CFTR modulator therapy effective in improving the quality of life of patients with cystic fibrosis who have one Phe508del allele?” Study Design: A
Avallone, Gia
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CFTR Modulator Therapy in the Treatment of Cystic Fibrosis as Compared to Placebo [PDF]
Objective: To assess the efficacy of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies including ivacaftor, tezacaftor/ivacaftor, and elexacaftor/tezacaftor/ivacaftor combinations in the improvement of lung function in ...
Doody, Rebecca Leigh +1 more
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Deciphering pediatric respiratory diseases using single-cell RNA sequencing [PDF]
The respiratory system comprises specialized cell types that maintain homeostasis and facilitate air conductance and gas exchange. Disease states often involve dysregulation of this balance, leading to changes in cell composition and molecular profiles ...
Loske, Jennifer
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Respiratory ResearchBackground 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 +7 more
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Medication adherence to CFTR modulators in patients with cystic fibrosis:a systematic review [PDF]
BACKGROUND: In the last decade, a fundamental shift in the treatment of cystic fibrosis (CF) took place due to the introduction of CF transmembrane conductance regulator (CFTR) modulators.
Breukelman, Anna J +5 more
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