Results 61 to 70 of about 4,770 (180)

Current prices versus minimum costs of production for CFTR modulators [PDF]

, 2022
BACKGROUND: While the clinical benefits of CFTR modulators are clear, their high prices render them inaccessible outside of the world's richest countries.
Fortunak, J, Guo, J, Hill, A, Wang, J, Zhang, J   +4 more
core   +1 more source

Elexacaftor–Tezacaftor–Ivacaftor Reduces Revision Sinus Surgery in People With Cystic Fibrosis

The Laryngoscope, EarlyView.
Elexacaftor–tezacaftor–ivacaftor (ETI) was associated with a significant decrease in the frequency and rate of endoscopic sinus surgeries in patients with cystic fibrosis. These findings suggest an improvement in CF‐related chronic rhinosinusitis outcomes following ETI and may influence future CF treatment decisions.
Amy Lin, Makanko Komara, Isaac Kistler, Zheng Hong Tan, Meredith Lind, Kavitha Kotha, Tendy Chiang   +6 more
wiley   +1 more source

Longitudinal profiling of the intestinal microbiome in children with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor

mBio
The intestinal microbiome influences growth and disease progression in children with cystic fibrosis (CF). Elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA), the newest pharmaceutical modulator for CF, restores the function of the pathogenic mutated CF ...
Seth A. Reasoner, Rachel Bernard, Adam Waalkes, Kelsi Penewit, Janessa Lewis, Andrew G. Sokolow, Rebekah F. Brown, Kathryn M. Edwards, Stephen J. Salipante, Maria Hadjifrangiskou, Maribeth R. Nicholson   +10 more
doaj   +1 more source

Longitudinal effects of elexacaftor/tezacaftor/ivacaftor on the oropharyngeal metagenome in adolescents with cystic fibrosis [PDF]

Background Triple modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI) improves lung function and impacts upon the respiratory microbiome in people with Cystic fibrosis (pwCF) with advanced lung disease. However, adolescents with cystic fibrosis (CF)
Gisler, Amanda, Hilty, Markus, Moeller, Alexander, Mostacci, Nadja, Steinberg, Ruth, Usemann, Jakob   +5 more
core   +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., MANEKA, S. LAKSHMI, SARAVANAKUMAR, R. T.   +2 more
core   +1 more source

Improved Quality of Life in Children With Cystic Fibrosis Who Received Transmembrane Conductance Regulator Modulators

Acta Paediatrica, EarlyView.
ABSTRACT Aim Children with cystic fibrosis (CF) face substantial daily treatment burdens and the effects of transmembrane conductance regulator modulators on these have not been sufficiently described. We evaluated changes in treatment burden after elexacaftor tezacaftor ivacaftor (ETI) was initiated.
Marcus Svedberg, Frida Lundqvist, Huda Abdulahi Östrand, Christina Krantz, Per Ekman, Henrik Imberg   +5 more
wiley   +1 more source

Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles

ERJ Open Research, 2019
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene (CFTR) that result in diminished quantity and/or function of the CFTR anion channel.
Jennifer L. Taylor-Cousar, Marcus A. Mall, Bonnie W. Ramsey, Edward F. McKone, Elizabeth Tullis, Gautham Marigowda, Charlotte M. McKee, David Waltz, Samuel M. Moskowitz, Jessica Savage, Fengjuan Xuan, Steven M. Rowe   +11 more
doaj   +1 more source

Molecular basis of cystic fibrosis and modern therapeutic approaches [PDF]

, 2019
Identifikacija molekularne povezanosti između genske nefunkcionalnosti CFTR gena te patofiziološke i kliničke pozadine cistične fibroze omogućila je razvoj suvremenih terapijskih modela koji su primarno utemeljeni na individualiziranom pristupu ...
Dragičević, Dorian
core   +2 more sources

Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study

Pharmaceuticals, 2022
The use of modulator drugs that target the Cystic Fibrosis transmembrane conductance regulator (CFTR) is the final frontier in the treatment of Cystic Fibrosis (CF), a genetic multiorgan disease.
Giuseppe Migliorisi, Mirella Collura, Francesca Ficili, Tiziana Pensabene, Dafne Bongiorno, Antonina Collura, Francesca Di Bernardo, Stefania Stefani   +7 more
doaj   +1 more source

Successful pregnancy in a cystic fibrosis patient with a severe impairment of lung function receiving Elexacaftor-Tezacaftor-Ivacaftor [case report]. [PDF]

, 2022
Before the arrival of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators women with CF and impaired lung function were experiencing a high risk of complications and mortality during and the years after pregnancy.
Balmpouzis, Zisis, Baud, David, Faure van Rossum, Annabelle, Koutsokera, Angela, Mazza Stalder, Jesica, Mitropoulou, Georgia, Panchaud, Alice   +6 more
core   +3 more sources