Results 91 to 100 of about 9,288 (216)

Effectiveness and safety of elexacaftor/tezacaftor/ivacaftor treatment in children aged 6‐11 years with cystic fibrosis in a real‐world setting

Pediatric Pulmonology
Elexacaftor‐tezacaftor‐ivacaftor (ETI) is a highly effective cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulating therapy for people with CF and at least one F508del variant.
V. Daccò, C. Rosazza, A. Mariani, C. Rizza, Nicolò Ingianni, Erica Nazzari, V. Terlizzi, Francesco Blasi, G. Alicandro   +8 more
semanticscholar   +1 more source

Impact of lumacaftor/ivacaftor on the bacterial and fungal respiratory pathogens in cystic fibrosis: a prospective multicenter cohort study in Sweden

Therapeutic Advances in Respiratory Disease
Background: A significant decline in pulmonary exacerbation rates has been reported in CF patients homozygous for F508del treated with lumacaftor/ivacaftor. However, it is still unclear whether this reduction reflects a diminished microbiological burden.
Mahasin Al Shakirchi, Kimmo Sorjonen, Lena Hjelte, Lena Klingspor, Peter Bergman, Petrea Ericson, Marcus Svedberg, Ulrika Lindberg, Christine Hansen, Isabelle de Monestrol   +9 more
doaj   +1 more source

The experience with elexacaftor/tezacaftor/ivacaftor + ivacaftor in adult patients with cystic fibrosis in the Omsk region

PULMONOLOGIYA
   Elexacaftor/tezacaftor/ivacaftor + ivacaftor, a drug modulating the cystic fibrosis transmembrane conductance regulator protein, is currently revolutionizing the management of patients with cystic fibrosis, particularly those with at least one F508del
N. Ovsyannikov, O. Bilevich, V. G. Berezhnoy, E. V. Romanovskaya, O. P. Minkovich, E. Barabanova, M. Karimov   +6 more
semanticscholar   +1 more source

Elexacaftor/tezacaftor/ivacaftor and mental health: A workshop report from the Cystic Fibrosis Foundation's Prioritizing Research in Mental Health working group.

Journal of Cystic Fibrosis
BACKGROUND This report summarizes the 2023 inaugural annual meeting of the Cystic Fibrosis Foundation's Prioritizing Research in Mental Health (PRIME) working group.
C. Bathgate, DA Fedele, E. Tillman, J. He, R. Everhart, LR Reznikov, Ff Liu, K. Kirby, K. Raffensperger, K. Traver, K. Riekert, SW Powers, AM Georgiopoulos   +12 more
semanticscholar   +1 more source

In utero and postnatal ivacaftor/lumacaftor therapy rescues multiorgan disease in CFTR-F508del ferrets

JCI Insight
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, with F508del being the most prevalent mutation.
I. Evans, Xingshen Sun, B. Liang, Amber R. Vegter, Lydia J. Guo, T. Lynch, Yan Zhang, Yulong Zhang, Y. Yi, Yu Yang, Zehua Feng, Soo Yeun Park, Amanita Shonka, Hannah McCumber, Lisi Qi, Peipei Wu, Guangming Liu, Allison Lacina, Kai Wang, K. Gibson-Corley, D. Meyerholz, D. Limoli, Bradley H. Rosen, Ziying Yan, Douglas J Bartels, John F. Engelhardt   +25 more
semanticscholar   +1 more source

Impact of elexacaftor-tezacaftor-ivacaftor in lung transplantation for cystic fibrosis in the United States

JHLT Open
Background: Cystic fibrosis (CF) is an autosomal recessive condition leading to progressive lung disease and often necessitating lung transplantation. Historically, CF has been one of the leading indications for lung transplants in the United States. The
Tahuanty A. Pena, MD, MS, Brittany Wright, PharmD, Kalpaj R. Parekh, MBBS, Julia Kleney-Tait, MD, PhD   +3 more
doaj   +1 more source

One year of treatment with elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis homozygous for the F508del mutation causes a significant increase in liver biochemical indexes

Frontiers in Molecular Biosciences
Introduction: Modulators of cystic fibrosis transmembrane conductance regulator mutated protein significantly improved the outcome of patients with cystic fibrosis (CF).
A. Castaldo, M. Gelzo, P. Iacotucci, A. Longobardi, G. Taccetti, V. Terlizzi, V. Carnovale   +6 more
semanticscholar   +1 more source

Extent of foetal exposure to maternal elexacaftor/tezacaftor/ivacaftor during pregnancy

British Journal of Pharmacology
Cystic fibrosis (CF) patients are living longer and healthier due to improved treatments, e.g. cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI), with treatment possibly occurring in ...
Danni Li, M. Donnelley, David Parsons, M. Habgood, E. Schneider-Futschik   +4 more
semanticscholar   +1 more source

Mitochondrial Fragmentation Induced by the CFTR Modulators Lumacaftor and Ivacaftor in Immortalized Cystic Fibrosis Cell Lines

Cells
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the CFTR gene, which encodes a cAMP-activated chloride channel essential for epithelial function.
Camila Dib, Pablo A. Iglesias González, María de los Ángeles Aguilar, Guillermo L. Taminelli, Tatiana Limpias del Valle, Nadia E. Nuñez, Analía G. Karadayian, Tomás A. Santa-Coloma, Ángel G. Valdivieso   +8 more
doaj   +1 more source

Editorial: Real-world experience with CFTR modulator therapy

Frontiers in Pharmacology, 2023
Burkhard Tümmler, Burkhard Tümmler, Pierre-Régis Burgel, Pierre-Régis Burgel   +3 more
doaj   +1 more source