Results 71 to 80 of about 12,467 (245)

P151 Pregnancies with elexacaftor/tezacaftor/ivacaftor in Argentina [PDF]

, 2023
E. Baran, L. Osken, N. Bergas Gimbernat
openalex   +1 more source

Bioactive Thymosin Alpha-1 Does Not Influence F508del-CFTR Maturation and Activity. [PDF]

, 2019
Deletion of phenylalanine 508 (F508del) in the cystic fibrosis transmembrane conductance regulator (CFTR) anion channel is the most frequent mutation causing cystic fibrosis (CF). F508del-CFTR is misfolded and prematurely degraded. Recently thymosin a-1 (
Armirotti, Andrea, Braccia, Clarissa, Cholon, Deborah M, Galietta, Luis JV, Gentzsch, Martina, Guidone, Daniela, Hanrahan, John W, Lukacs, Gergely L, Matthes, Elizabeth, Pedemonte, Nicoletta, Phuan, Puay-Wah, Tomati, Valeria, Veit, Guido, Verkman, Alan S   +13 more
core  

Opportunities of patient‐derived organoids in drug development

British Journal of Pharmacology, Volume 183, Issue 4, Page 939-944, February 2026.
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

A pediatric cystic fibrosis arthropathy case who responded to Elexacaftor/Tezacaftor/Ivacaftor therapy [PDF]

, 2023
Meliksah Arslan, Zeynep Bahadır, Matthew L. Basiaga, Sarah Chalmers, Nadir Demirel   +4 more
openalex   +1 more source

The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel [PDF]

, 2014
The cystic fibrosis transmembrane conductance regulator (CFTR) plays a pivotal role in fluid and electrolyte movements across ducts and tubes lined by epithelia.
Cai, Zhiwei, Cami-Kobeci, Gerta, Husbands, Stephen, Khuituan, Pissared, Li, Hongyu, Liu, Jia, Sheppard, David, Wang, Yiting   +7 more
core   +2 more sources

Pharmacogenomics in clinical practice: Biomarker information in Brazilian drug labels

British Journal of Clinical Pharmacology, Volume 92, Issue 1, Page 109-118, January 2026.
This review examines the PGx annotations in package inserts (bulas in Brazilian Portuguese) approved by ANVISA, the Brazilian Health Regulatory Agency, for 19 gene–drug pairs with strong or moderate recommendations for initial dosing alteration in the CPIC (Clinical Pharmacogenetic Implementation Consortion) guidelines and PGx testing required or ...
Guilherme Suarez‐Kurtz
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

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

Strategies against nonsense: oxadiazoles as translational readthrough-inducing drugs (TRIDs) [PDF]

, 2019
This review focuses on the use of oxadiazoles as translational readthrough-inducing drugs (TRIDs) to rescue the functional full-length protein expression in mendelian genetic diseases caused by nonsense mutations.
Campofelice A., Di Leonardo A., Lentini L., Melfi R., Pace A., Pibiri I., Tutone M.   +6 more
core   +1 more source

Successes and Challenges: Inhaled Treatment Approaches Using Magnetic Nanoparticles in Cystic Fibrosis

Magnetochemistry, 2020
Magnetic nanoparticles have been largely applied to increase the efficacy of antibiotics due to passive accumulation provided by enhancing permeability and retention, which is essential for the treatment of lung infections. Recurring lung infections such
Marsha Tan, Felisa Reyes-Ortega, Elena K. Schneider-Futschik   +2 more
doaj   +1 more source