Results 61 to 70 of about 9,731 (210)

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  

Correcting for tissue nitrogen excretion in multiple breath washout measurements. [PDF]

, 2017
Nitrogen excreted from body tissues impacts the calculation of multiple breath nitrogen washout (MBWN2) outcomes. The aim of this study was to determine the effect of tissue N2 on MBWN2 outcomes in both healthy subjects and patients with CF and to assess
Jensen, Renee, Kane, Mica, McDonald, Reginald, Ratjen, Felix, Rayment, Jonathan H., Stanojevic, Sanja   +5 more
core   +3 more sources

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

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

Consequences of cystic fibrosis transmembrane regulator mutations on inflammatory cells [PDF]

, 2016
Recent studies in cystic fibrosis (CF) transmembrane regulator (CFTR) mutations and function have shed light on its involvement in disease progression. The extent of cell and tissue distribution of CFTR facilitates systemic dysfunction of ion transport ...
Castro, Guillermo Raul, Grumelli, Sandra, Islan, German Abel   +2 more
core   +1 more source

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, Volume 36, Issue 36, 4 May 2026.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

Children, 2023
Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic ...
Nikoletta Kapouni, Maria Moustaki, Konstantinos Douros, Ioanna Loukou   +3 more
doaj   +1 more source

Farmacogenética en Fibrosis Quística. [PDF]

, 2017
La fibrosis quística (a partir de ahora referida como FQ) fue descrita por primera vez en 1936 como una entidad separada. También recibía el nombre de “mucoviscidosis” debido a la acumulación de espesas secreciones mucosas que bloqueaban las vías aéreas,
Oraá Pérez, Javier
core  

Are new models needed to optimize the utilization of new medicines to sustain healthcare systems? [PDF]

, 2014
Medicines have made an appreciable contribution to improving health. However, even high-income countries are struggling to fund new premium-priced medicines. This will grow necessitating the development of new models to optimize their use.
Acurcio, Francisco, Alkan, Ali, Brzezinska, Anna, Bucsics, Anna, Campbell, Stephen M, Czeczot, Jadwiga, de Bruyn, Winnie, Diogene, Eduardo, Eriksson, Irene, Finlayson, Alexander E, Fürst, Jurij, Garuoliene, Kristina, Godman, Brian, Gray, Andy, Guerra Júnior, Augusto, Gulbinovič, Jolanta, Gustafsson, Lars L, Jan, Saira, Jayathissa, Sisira, Joppi, Roberta, Kalaba, Marija, Magnisson, Einar, Malmström, Rickard E, McCullagh, Laura, Miikkulainen, Kaisa, Ofierska-Sujkowska, Gabriela, Pedersen, Hanne Bak, Selke, Gisbert, Sermet, Catherine, Spillane, Susan, Supian, Azuwana, Timoney, Angela, Truter, Ilse, Vien, Low Ee, Vlahović-Palčevski, Vera, Vural, Elif H, Wale, Janet, Władysiuk, Magdałene, Yusof, Faridah Aryani Md, Zeng, Wenjie   +39 more
core   +1 more source