Results 61 to 70 of about 4,639 (201)

Respiratory Oscillometry and Multimodal Lung Function Assessment of Elexacaftor/Tezacaftor/Ivacaftor Response in Children and Young Adults With Cystic Fibrosis

Pediatric Pulmonology, Volume 61, Issue 2, February 2026.
ABSTRACT Background To date, there is no data on forced oscillation technique (FOT) as a tool to evaluate elexacaftor/tezacaftor/ivacaftor (ETI) modulator in young patients with cystic fibrosis (pwCF). Objective Our study aimed to assess the effect of a 6‐month ETI treatment on FOT parameters and compare it to the effect on spirometry and nitrogen ...
Christos Kogias, Athina Sopiadou, Sotirios Fouzas, Petrina Vantsi, Elissavet‐Anna Chrysochoou, Evangelia Desli, Maria Galogavrou, Nikolaos Gasparis, Elpis Hatziagorou   +8 more
wiley   +1 more source

Modeling long-term health outcomes of patients with cystic fibrosis homozygous for treated with lumacaftor/ivacaftor

Therapeutic Advances in Respiratory Disease, 2019
Background: Lumacaftor/ivacaftor combination therapy is efficacious and generally safe for patients with cystic fibrosis (CF) homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation.
Jaime L. Rubin, Lasair O’Callaghan, Christopher Pelligra, Michael W. Konstan, Alexandra Ward, Jack K. Ishak, Conor Chandler, Theodore G. Liou   +7 more
doaj   +1 more source

Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype

Molecular Genetics & Genomic Medicine, 2021
Background New drugs that target the basic defect in cystic fibrosis (CF) patients may now be used in a large number of patients carrying responsive mutations. Nevertheless, further research is needed to extend the benefit of these treatments to patients
Vito Terlizzi, Felice Amato, Chiara Castellani, Beatrice Ferrari, Luis J. V. Galietta, Giuseppe Castaldo, Giovanni Taccetti   +6 more
doaj   +1 more source

Pediatric population with cystic fibrosis in the centre of Portugal: candidates for new therapies

Jornal de Pediatria, 2022
Objectives: Cystic fibrosis (CF) is a severe autosomal recessive disease that results from mutations in a gene encoding the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, a chloride channel.
Juliana Roda, Teresa Teixeira, Iris AI Silva, Teresa Reis Silva, Ricardo Ferreira, Margarida D. Amaral, Guiomar Oliveira   +6 more
doaj   +1 more source

Effects of cAMP and CFTR modulation on apical fluid pH in human airway Calu‐3 cells

Physiological Reports, Volume 14, Issue 3, February 2026.
Abstract The airway epithelium serves as the first line of defense against inhaled insults present in the external environment by acting as a physical barrier and through host defense mechanisms. Proper maintenance of these host defense mechanisms relies on the regulation of airway surface liquid (ASL) composition and properties, a process that is ...
Jenny P. Nguyen, Nadia Milad, Jeremy A. Hirota   +2 more
wiley   +1 more source

Structure-activity analysis of a CFTR channel potentiator: Distinct molecular parts underlie dual gating effects. [PDF]

, 2014
The cystic fibrosis (CF) transmembrane conductance regulator (CFTR) is a member of the ATP-binding cassette transporter superfamily that functions as an epithelial chloride channel. Gating of the CFTR ion conduction pore involves a conserved irreversible
Ahern, Al-Shawi, Aleksandrov, Basso, Beáta Töröcsik, Boyle, Chan, Cheng, Cholon, Csanády, Csanády, Csanády, Csanády, Csanády, Dalton, Dean, Gadsby, Gunderson, Hollenstein, Hwang, Jih, Jih, Kopeikin, Linsdell, Linsdell, László Csanády, Miki, Ramjeesingh, Ramsey, Riordan, Tsai, Van Goor, Van Goor, Veit, Vergani, Vergani, Wang, Wangemann   +37 more
core   +1 more source

Farmacogenética en Fibrosis Quística. [PDF]

, 2017
La fibrosis quística (a partir de ahora referida como FQ) fue descrita por primera vez en 1936 como una entidad separada. También recibía el nombre de “mucoviscidosis” debido a la acumulación de espesas secreciones mucosas que bloqueaban las vías aéreas,
Oraá Pérez, Javier
core  

Predictive factors for lumacaftor/ivacaftor clinical response [PDF]

Journal of Cystic Fibrosis, 2019
Ivacaftor-lumacaftor combination therapy corrects the F508 del-CFTR mutated protein which causes Cystic Fibrosis. The clinical response of the patients treated with the combination therapy is highly variable. This study aimed to determine factors involved in the individual's response to lumacaftor-ivacaftor therapy.Sweat test was assessed at baseline ...
Masson, Alexandra, Schneider-Futschik, Elena K., Baatallah, Nesrine, Nguyen-Khoa, Thao, Girodon, Emmanuelle, Hatton, Aurélie, Flament, Thomas, Le Bourgeois, Muriel, Chedevergne, Frederique, Bailly, Céline, Kyrilli, Sylvia, Achimastos, Diane, Hinzpeter, Alexandre, Edelman, Aleksander, Sermet-Gaudelus, Isabelle   +14 more
openaire   +2 more sources

The validated French CFAbd‐Score reveals a lower burden of gastrointestinal symptoms in patients on Elexacaftor/Tezacaftor/Ivacaftor

Journal of Pediatric Gastroenterology and Nutrition, Volume 82, Issue 1, Page 24-32, January 2026.
Abstract Objectives Multiorgan abdominal involvement is a hallmark of Cystic fibrosis (CF). The CFAbd‐Score© is the first CF‐specific gastrointestinal patient reported outcome‐measure (PROM) developed following FDA‐guidelines. The PROM has proved to sensitively differentiate people with CF (pwCF) from healthy controls (HC).
Isabelle Sermet‐Gaudelus, Pauline Sadrieh, Anne Sophie Bonnel, Raphael Enaud, Nathalie Wizla, Jeanne Languepin, Ambre Nangui, Audrey Deelawon, Johanna Rave, Samira Dabelow, Anton Barucha, Carlos Zagoya, Franziska Duckstein, Jochen Georg Mainz   +13 more
wiley   +1 more source

Preclinical evaluation of the epithelial sodium channel inhibitor BI 1265162 for treatment of cystic fibrosis

ERJ Open Research, 2020
Background Epithelial sodium channel (ENaC) is an important regulator of airway surface liquid volume; ENaC is hyperactivated in cystic fibrosis (CF). ENaC inhibition is a potential therapeutic target for CF.
Peter Nickolaus, Birgit Jung, Juan Sabater, Samuel Constant, Abhya Gupta   +4 more
doaj   +1 more source