Results 41 to 50 of about 7,354 (172)

Opportunities of patient‐derived organoids in drug development

British Journal of Pharmacology, EarlyView.
Various model systems are utilised during drug development starting from basic research, moving to preclinical research and development for clinical applications in order to identify new drugs to improve human health. However, there are characteristics of humans that are not captured by established models.
Antonia Büning, Elena Reckzeh
wiley   +1 more source

Efectividad de ivacaftor en vida real en niños con fibrosis quística y mutación G551D

Anales de Pediatría, 2019
Resumen: Introducción: Ivacaftor es un potenciador de la proteína reguladora de la conductancia transmembrana de la fibrosis quística (CFTR) que ha demostrado en ensayos clínicos mejoría del estado nutricional y la función pulmonar de pacientes con ...
David Gomez-Pastrana, Chinedu Nwokoro, Mike McLean, Sarah Brown, Nanna Christiansen, Caroline S. Pao   +5 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

Pre-Processing a Polymer Blend into a Polymer Alloy by KinetiSol Enables Increased Ivacaftor Amorphous Solid Dispersion Drug Loading and Dissolution

Biomedicines, 2023
This study compares the effects of pre-processing multiple polymers together to form a single-phase polymer alloy prior to amorphous solid dispersion formulation.
Stephen A. Thompson, Daniel A. Davis, Dave A. Miller, Sandra U. Kucera, Robert O. Williams   +4 more
doaj   +1 more source

Lumacaftor/ivacaftor in cystic fibrosis: effects on glucose metabolism and insulin secretion [PDF]

, 2021
Carla Colombo, Andrea Foppiani, Arianna Bisogno, Simone Gambazza, Valeria Daccò, Erica Nazzari, Alessandro Leone, A. Giana, Andrea Mari, Alberto Battezzati   +9 more
openalex   +1 more source

In silico, in vitro and ex vivo characterization of cystic fibrosis transmembrane conductance regulator pathogenic variants localized in the fourth intracellular loop and their rescue by modulators

British Journal of Pharmacology, Volume 182, Issue 24, Page 6063-6080, December 2025.
Background and Purpose Cystic fibrosis (CF) is due to loss‐of‐function variants of the CF transmembrane conductance regulator (CFTR) channel. The most effective treatment for people with CF carrying the F508del mutation is the triple combination of elexacaftor–tezacaftor–ivacaftor (ETI).
Emanuela Pesce, Valeria Tomati, Valeria Capurro, Mariateresa Lena, Cristina Pastorino, Miro Astore, Serdar Kuyucak, Benoit Chevalier, Elvira Sondo, Federico Cresta, Alice Dighero, Vito Terlizzi, Cristina Fevola, Stefano Costa, Maria Cristina Lucanto, Valeria Daccò, Laura Claut, Francesca Ficili, Benedetta Fabrizzi, Renata Bocciardi, Federico Zara, Carlo Castellani, Luis J. V. Galietta, Shafagh A. Waters, Alexandre Hinzpeter, Nicoletta Pedemonte   +25 more
wiley   +1 more source

Beyond Carrier Status: CFTR Heterozygosity as an Overlooked Clinical Risk Factor for Pancreatitis

Clinical Genetics, Volume 108, Issue 6, Page 726-730, December 2025.
CFTR carrier status detected via prenatal screening is associated with increased pancreatitis risk in women, but clinical recognition remains low. Integrating carrier results into electronic health records may enhance personalized care and aid in early diagnosis. ABSTRACT This study assessed the effect of CFTR pathogenic variant status, detected during
Lucas D. Richter, Douglas M. Ruderfer, Josh F. Peterson, Lisa A. Bastarache   +3 more
wiley   +1 more source

Home Microbiological Sampling in a Pediatric Cystic Fibrosis Population: Pandemic Implementation and Ongoing Use

Pediatric Pulmonology, Volume 60, Issue 11, November 2025.
ABSTRACT Background During the coronavirus disease 2019 (COVID19) pandemic, restrictions on in‐person care threatened to disrupt in‐clinic airway sampling for microbiological surveillance, a vital aspect of cystic fibrosis (CF) care. In response, institutions developed home airway sampling strategies to allow continued guidelines‐based microbiological ...
Valerie Swanston, Jeffrey N. Bone, Melissa Richmond, David Goldfarb, Jonathan H. Rayment   +4 more
wiley   +1 more source

Physiologically‐based pharmacokinetic modeling of the drug–drug interaction between ivacaftor and lefamulin in cystic fibrosis patients

CPT: Pharmacometrics & Systems Pharmacology
Lefamulin is being evaluated as a treatment for bacterial exacerbations in cystic fibrosis (CF). Ivacaftor is approved for the treatment of patients with CF.
Magali Amiel, Alice Ke, Steven P. Gelone, Hannah M. Jones, Wolfgang Wicha   +4 more
doaj   +1 more source

Analysis of Ivacaftor drug approval for cystic fibrosis patients with gating mutations. [PDF]

The Young Researcher, 2021
Cystic fibrosis is an inherited monogenetic disorder that leads to chronic respiratory and lung infections. These infections result in decreased quality of life in patients.
Nemirajaiah, S.
doaj