Results 81 to 90 of about 9,731 (210)

Integrating Human Intestinal Organoids into FDA's New Approach Methodologies for Drug Discovery

Advanced Science, Volume 13, Issue 20, 9 April 2026.
Illustration summarizes how human intestinal organoids (HIOs) are becoming transformative in preclinical research. Preclinical drug discovery pipelines often rely on animal models for ADMET studies, even though interspecies ADME gaps, poor external validity, and high attrition rates are common.
Debarun Patra, Ibrahim M. Sayed, Souhrid Mukherjee, Honit Piplani, Aida Habtezion, Michael J. Rosen, Jospeh C. Wu   +6 more
wiley   +1 more source

Efficient, non‐toxic anion transport by synthetic carriers in cells and epithelia [PDF]

, 2016
Transmembrane anion transporters (anionophores) have potential for new modes of biological activity, including therapeutic applications. In particular they might replace the activity of defective anion channels in conditions such as cystic fibrosis ...
A Vargas Jentzsch, Anthony P. Davis, AP Davis, AS Verkman, AV Koulov, B Shen, BA McNally, BC Pressman, BD de Grenu, BW Ramsey, CA Lipinski, CE Wainwright, CJ Dutton, CW Jiang, DA Stoltz, David N. Sheppard, DN Sheppard, DP Wallace, EWFW Alton, FM Ashcroft, G McLachlan, GR Cutting, GW Gokel, H Li, H Valkenier, H Valkenier, Hazel A. Sparkes, Hennie Valkenier, Hongyu Li, JA Cooper, James A. Cooper, JL Sessler, JL Sessler, JP Clare, JR Broughman, JR Broughman, JT Davis, JT Davis, LJV Galietta, Luke W. Judd, LVJ Galietta, MA Mall, N Busschaert, N Busschaert, Ondřej Jurček, PA Gale, Peter R. Brotherhood, PR Brotherhood, S Hussain, Sabir Hussain, SJ Edwards, SJ Moore, SK Ko, V Sidorov, X Li   +54 more
core   +2 more sources

3D Automated Segmentation of Bronchial Abnormalities on Ultrashort Echo Time MRI: A Quantitative MR Outcome in Cystic Fibrosis

Journal of Magnetic Resonance Imaging, Volume 63, Issue 4, Page 1190-1200, April 2026.
ABSTRACT Background Cystic fibrosis (CF) monitoring relies on computed tomography (CT), but ultra‐short echo time MRI (UTE‐MRI) offers a radiation‐free alternative. However, its clinical adoption is hindered by the laborious and subjective manual analysis, which prevents standardized quantification of bronchial abnormalities.
Amel Imene Hadj Bouzid, Ilyes Benlala, Baudouin Denis de Senneville, Fabien Baldacci, Julie Macey, Wadie Benhassen, Pan Su, Stephanie Bui, Maeva Zysman, Aurelien Bustin, Patrick Berger, Gael Dournes   +11 more
wiley   +1 more source

A new familial form of a late-onset, persistent hyperinsulinemic hypoglycemia of infancy caused by a novel mutation in KCNJ11. [PDF]

, 2017
The ATP-sensitive potassium channel (KATP) functions as a metabo-electric transducer in regulating insulin secretion from pancreatic β-cells. The pancreatic KATP channel is composed of a pore-forming inwardly-rectifying potassium channel, Kir6.2, and a ...
Ferrara, Christine, German, Michael, Gitelman, Stephen, Long, Roger, Yang, Shi-Bing, Yang, Yen-Yu   +5 more
core   +1 more source

Patient‐Derived Intestinal Organoids in the Global Cystic Fibrosis Landscape

Pediatric Pulmonology, Volume 61, Issue 4, April 2026.
ABSTRACT Cystic fibrosis (CF) care has advanced rapidly, yet diagnosis and inclusion in patient registries remain severely limited in low‐ and middle‐income countries (LMICs). Barriers include restricted newborn screening, limited availability of sweat chloride testing, and underrepresentation of non‐European CFTR variants in standard panels.
Suzanne Kroes, Jennifer L. Taylor‐Cousar, Marco Zampoli, Bülent Karadag, Cornelis K. van der Ent, Karin M. de Winter – de Groot, Jeffrey M. Beekman, Sacha Spelier   +7 more
wiley   +1 more source

Ivacaftor ameliorates mucus burden, bacterial load, and inflammation in acute but not chronic P. aeruginosa infection in hG551D rats

Respiratory Research
Background Newly approved highly effective modulation therapies (HEMT) have been life-changing for people with CF. Although these drugs have resulted in significant improvements in lung function and exacerbation rate, bacterial populations in the lung ...
Johnathan D. Keith, Mikayla Murphree-Terry, Gretchen Bollar, Ashley M. Oden, Ian H. Doty, Susan E. Birket   +5 more
doaj   +1 more source

Health state utility data in Cystic Fibrosis: A systematic review [PDF]

, 2019
Introduction: Cystic fibrosis (CF) is a life-limiting, hereditable condition, with the highest prevalence in Europe. CF treatments have led to improvements in clinical symptoms, disease management and decelerated disease progression.
Archangelidi, Olga, Bhadhuri, Arjun, Bilton, Diana, Carr, Siobhan, Mohindru, Bishal, Sach, Tracey, Turner, David, Whitty, Jennifer   +7 more
core   +1 more source

Ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation [PDF]

European Respiratory Review, 2013
Cystic fibrosis (CF) is an autosomal recessive lethal disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that encodes for CFTR, an epithelial cell-surface expressed protein responsible for the transport of ...
Isabelle Sermet-Gaudelus
doaj  

Management of Infants Exposed to Prenatal and Postnatal Elexacaftor/Tezacaftor/Ivacaftor: A Cross‐Sectional Survey

Health Science Reports, Volume 9, Issue 5, May 2026.
Haley Haskett, Clement L. Ren
wiley   +1 more source

Impact of the CFTR-potentiator ivacaftor on airway microbiota in cystic fibrosis patients carrying a G551D mutation.

PLoS ONE, 2015
BackgroundAirway microbiota composition has been clearly correlated with many pulmonary diseases, and notably with cystic fibrosis (CF), an autosomal genetic disorder caused by mutation in the CF transmembrane conductance regulator (CFTR).
Cédric Bernarde, Marlène Keravec, Jérôme Mounier, Stéphanie Gouriou, Gilles Rault, Claude Férec, Georges Barbier, Geneviève Héry-Arnaud   +7 more
doaj   +1 more source