Impact of CFTR Modulators on Beta-Cell Function in Children and Young Adults with Cystic Fibrosis [PDF]
, 2022 Background: To date, no consistent data are available on the possible impact of CFTR modulators on glucose metabolism. The aim of this study was to test the hypothesis that treatment with CFTR modulators is associated with an improvement in the key ...
Bonadonna R. C. +14 more
core +1 more source
Clinical Case Reports, 2021 Elexacaftor/tezacaftor/ivacaftor (ETI) is a cystic fibrosis (CF) transmembrane regulator (CFTR) modulator. It is known to be efficacious in stable patients with severe pneumopathy, but there are few data concerning its effectiveness during acute ...
Donatello Salvatore +4 more
doaj +1 more source
Pharmaceuticals, 2023 Cystic fibrosis (CF) is a potentially fatal monogenic disease that causes a progressive multisystemic pathology. Over the last decade, the introduction of CF transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has profoundly
Mafalda Bacalhau +5 more
doaj +1 more source
WS05.05 Randomised withdrawal of hypertonic saline in those with lower lung function after receiving elexacaftor/tezacaftor/ivacaftor; a sub-study of the SIMPLIFY Trial [PDF]
, 2023 David P. Nichols +9 more
openalex +1 more source
Qualitative and quantitative evaluation of computed tomography changes in adults with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor: a retrospective observational study [PDF]
, 2023 Introduction: The availability of highly effective triple cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapy with elexacaftor-tezacaftor-ivacaftor (ETI) has improved pulmonary outcomes and quality of life of people ...
Dettmer, Sabine +8 more
core +1 more source
Clinical Transplantation, Volume 40, Issue 1, January 2026.ABSTRACT Background: In pediatric lung transplantation, the ability to estimate the projected waiting time could be particularly helpful for candidates and physicians. A score to predict waiting time has yet to be developed in this field. The purpose of this study was to create a score that predicts waitlist time for pediatric patients who are listed ...
Jose Ramirez +2 more
wiley +1 more source
Genetics and Molecular Biology, 2021 This is a descriptive cross-sectional study that aims to determine the distribution of the CFTR causing variant in a group of patients at a cystic fibrosis (CF) center in southern Brazil, as well as to describe causing variants that are treatable with ...
Eliandra da Silveira Lima +3 more
doaj +1 more source
The effect of elexacaftor/tezacaftor/ivacaftor on non-pulmonary symptoms in adults with cystic fibrosis [PDF]
, 2023 Sarah Allgood +5 more
openalex +1 more source
DEVELOPMENT AND VALIDATION OF STABILITY-INDICATING RP-UPLC METHOD FOR THE SIMULTANEOUS ESTIMATION OF TEZACAFTOR AND IVACAFTOR IN FORMULATIONS [PDF]
, 2020 Objective: Aim of the present research work was to develop a sensitive, rapid and accurate, stability-indicating RP-UPLC method for the simultaneous estimation of tezacaftor and ivacaftor in formulations.
ANJANA, C. H. K. V. L. S. N. +2 more
core +1 more source
Beyond the Surface: Novel Therapy Approach for Pancreatitis in the Setting of CFTR Dysfunction
Case Reports in Gastrointestinal Medicine, Volume 2026, Issue 1, 2026.Objectives Despite cystic fibrosis transmembrane conductance regulator (CFTR) proteins being present throughout the entire body and organ systems, typical presentation of cystic fibrosis (CF) involves lung disease. We report a series of individuals that were referred to our CF clinic for evaluation following a diagnosis of chronic pancreatitis or acute
Brittany A. Wright +4 more
wiley +1 more source