Results 71 to 80 of about 1,459,532 (261)

Strategies to investigate the mechanism of action of CFTR modulators [PDF]

Journal of Cystic Fibrosis, 2004
The malfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel is associated with a wide spectrum of disease. In the search for modulators of CFTR, pharmaceutical agents have been identified that (i) act indirectly by regulating the protein kinases and phosphatases, which control CFTR, and (ii) interact directly with CFTR.
Cai, Zhiwei, Scott-Ward, Toby S., Li, Hongyu, Schmidt, André, Sheppard, David N.   +4 more
openaire   +2 more sources

Uncovering Cystic Fibrosis Carrier: Insights From a Heterozygous CFTR‐F508del Rabbit Model

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) is a heterogeneous inflammatory disorder frequently associated with impaired mucociliary clearance and bacterial infection. Individuals carrying a single cystic fibrosis transmembrane conductance regulator (CFTR) mutation exhibit partial CFTR dysfunction and are increasingly recognized as being at risk ...
Do‐Yeon Cho, Alexis E. McFeely, Daniel Skinner, Shaoyan Zhang, Justin Hedvat, Weslee Ping‐ay, Daniel M. Beswick, Justin H. Turner, Bradford A. Woodworth, Jie Xu   +9 more
wiley   +1 more source

Allergic Bronchopulmonary Aspergillosis (ABPA) in the Era of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators

Journal of Fungi
Allergic bronchopulmonary aspergillosis (ABPA) is a hypersensitivity disease caused by Aspergillus fumigatus (Af), prevalent in persons with cystic fibrosis (CF) or asthma.
Paulami Chatterjee, Carson Tyler Moss, Sarah Omar, Ekroop Dhillon, Carlos Daniel Hernandez Borges, Alan C. Tang, David A. Stevens, Joe L. Hsu   +7 more
doaj   +1 more source

Q1291H-CFTR molecular dynamics simulations and ex vivo theratyping in nasal epithelial models and clinical response to elexacaftor/tezacaftor/ivacaftor in a Q1291H/F508del patient

Frontiers in Molecular Biosciences, 2023
Background: Cystic fibrosis (CF) is caused by a wide spectrum of mutations in the CF transmembrane conductance regulator (CFTR) gene, with some leading to non-classical clinical presentations.
Katelin M. Allan, Katelin M. Allan, Katelin M. Allan, Miro A. Astore, Egi Kardia, Egi Kardia, Egi Kardia, Sharon L. Wong, Sharon L. Wong, Sharon L. Wong, Laura K. Fawcett, Laura K. Fawcett, Laura K. Fawcett, Laura K. Fawcett, Jessica L. Bell, Jessica L. Bell, Simone Visser, Po-Chia Chen, Renate Griffith, Adam Jaffe, Adam Jaffe, Adam Jaffe, Sheila Sivam, Orazio Vittorio, Orazio Vittorio, Serdar Kuyucak, Shafagh A. Waters, Shafagh A. Waters, Shafagh A. Waters, Shafagh A. Waters   +29 more
doaj   +1 more source

Determining the Minimal Clinically Important Difference of the 40‐Item Smell Identification Test in People With Cystic Fibrosis

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) are highly prevalent among people with cystic fibrosis (PwCF) and negatively impact quality of life. The 40‐item Smell Identification Test (SIT) is widely used to assess psychophysical olfaction, but a CF‐specific minimal clinically important difference (MCID) has not been ...
Eugene Oh, Jessa E. Miller, Michelle J. Lee, Anna Zemke, David Baraghoshi, Matthew J. Strand, Jeremiah A. Alt, Todd E. Bodner, Michael Chang, Naweed I. Chowdhury, Patricia H. Eshaghian, Anne E. Getz, Jennifer L. Goralski, David A. Gudis, Peter H. Hwang, Ashoke Khanwalkar, Adam J. Kimple, Jivianne T. Lee, Douglas A. Li, Jess C. Mace, Jayakar V. Nayak, Jonathan B. Overdevest, Zara Patel, Daniella K. Safatian, Rodney J. Schlosser, Brent Senior, Amanda L. Stapleton, Timothy L. Smith, Zachary M. Soler, Jeffrey D. Suh, Grant A. Turner, Marilene B. Wang, Milene T. Saavedra, Jennifer L. Taylor‐Cousar, Daniel M. Beswick   +34 more
wiley   +1 more source

CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare CFTR Genotypes [PDF]

, 2023
Cystic fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene. The combination of the CFTR modulators elexacaftor, tezacaftor, and ivacaftor (ETI) enables the effective rescue of CFTR function in ...
Muilwijk, Danya, Paul J. Niemöller, Hettie M. Janssens, van der Ent, Cornelis K., Natascha D. A. Nieuwenhuijze, Bierlaagh, Marlou C., Nieuwenhuijze, Natascha D.A., Janssens, Hettie M., Marlou C. Bierlaagh, Sam F. B. van Beuningen, Juliet W. Lefferts, Niemöller, Paul J., Verburg, Tibo F., Beekman, Jeffrey M., Danya Muilwijk, van Beuningen, Sam F.B., Cornelis K. van der Ent, Lefferts, Juliet W., Kroes, Suzanne, Heleen N. Sonneveld van Kooten, Tibo F. Verburg, Jeffrey M. Beekman, Sonneveld van Kooten, Heleen N., Suzanne Kroes   +23 more
core   +1 more source

CFTR Modulator Therapy with Lumacaftor/Ivacaftor Alters Plasma Concentrations of Lipid-Soluble Vitamins A and E in Patients with Cystic Fibrosis

Antioxidants, 2021
Rationale: Cystic fibrosis (CF), caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, leads to impaired pancreatic function and therefore reduced intestinal absorption of lipids and fat-soluble vitamins especially ...
O. Sommerburg, S. Hämmerling, S. Schneider, J. Okun, C. Langhans, Patricia Leutz-Schmidt, M. Wielpütz, W. Siems, S. Gräber, M. Mall, M. Stahl   +10 more
semanticscholar   +1 more source

Optimization of pharmaceutical research and development by early‐phase assessment of investigational medicinal products

British Journal of Clinical Pharmacology, EarlyView.
Small and mid‐sized pharmaceutical innovators often have limited in‐house health economics and market access expertise, and may struggle to align development strategies of investigational medicinal products with health system needs and payer expectations.
Zoltán Kaló, Attila Imre, Gábor Varga, Sándor Kovács, György Tímár, Balázs Nagy   +5 more
wiley   +1 more source

Effects of CFTR Modulators on Pseudomonas aeruginosa Infections in Cystic Fibrosis

Infectious Disease Reports
Background: Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Camelia Corina Pescaru, Alexandru Florian Crișan, Adelina Marițescu, Vlad Cărunta, Monica Marc, Ștefan Dumitrache-Rujinski, Sorina Laitin, Cristian Oancea   +7 more
doaj   +1 more source

Precision medicine in paediatrics: Progress and priorities

British Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain, Claire Booth, Joseph F. Standing, Charlotte I. S. Barker   +3 more
wiley   +1 more source