Results 101 to 110 of about 6,598 (226)

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

One year effect of tezacaftor and ivacaftor on functional exercise capacity and muscle strength in people with cystic fibrosis

Heliyon
Background: The 1-min sit-to-stand test (1STST) is a practical tool to evaluate physical capacity. The aim of this study was to assess the impact of tezacaftor and ivacaftor on functional exercise capacity, muscle strength and symptoms in people with ...
Aubriot Anne-Sophie, Morgane Penelle, Gonçalvès Clémence, Silvia Berardis, Christophe Goubau, Gregory Reychler, Sophie Gohy   +6 more
doaj   +1 more source

Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with Cystic Fibrosis following liver transplantation:A systematic review [PDF]

BACKGROUND & AIMS: Cystic Fibrosis (CF) liver disease progresses to liver failure requiring transplantation in about 3 % of patients, 0.7 % of CF patients are post liver transplant.
Brugha, Rossa, Indolfi, Giuseppe, Terlizzi, Vito, Testa, Ilaria, Verkade, Henkjan J   +4 more
core   +1 more source

Towards personalized medicine in cystic fibrosis:Focusing on clinical pharmacology of CFTR modulators [PDF]

This thesis aimed to improve understanding of the clinical pharmacology of CFTR modulators in children with cystic fibrosis (CF) using real-world data, thereby supporting progress towards personalized medicine.To enable pharmacokinetic analyses, two ...
Vonk, S.E.M.
core   +2 more sources

Urticaria multiforme-like eruption due to a novel agent elexacaftor/tezacaftor/ivacaftor in a pediatric patient with cystic fibrosis

JAAD Case Reports, 2021
Rebecca H. Goldberg, BS, Natalie H. Matthews, MD, MPhil, Alexandra C. Hristov, MD, Frank Wang, MD   +3 more
doaj   +1 more source

Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor

Molecules
The monogenic rare disease Cystic Fibrosis (CF) is caused by mutations in the gene encoding the CF transmembrane conductance (CFTR) protein, an anion channel expressed at the apical plasma membrane of epithelial cells.
Filipa C. Ferreira, Camilla D. Buarque, Miquéias Lopes-Pacheco   +2 more
doaj   +1 more source

564 Effects of elexacaftor-tezacaftor-ivacaftor modulator use during cystic fibrosis pregnancies on maternal and neonatal outcomes [PDF]

, 2023
A. Dau, H. Klasna, M. Stirek, M. Jackson, Jesse Wells, M. Timmerman, C. Hanson   +6 more
openalex   +1 more source

Tezacaftor is a direct inhibitor of sphingolipid delta-4 desaturase enzyme (DEGS) [PDF]

Background: We recently demonstrated that 48 h exposure of primary human bronchial epithelial (hBE) cells, obtained from both CF (F508del homozygous) and non-CF subjects, to the triple drug combination Elexacaftor/Tezacaftor/Ivacaftor (ETI) results in a ...
Armirotti, Andrea, Aureli, Massimo, Bandiera, Tiziano, Bassi, Rosaria, Bertorelli, Rosalia, Bertozzi, Sine Mandrup, Capurro, Valeria, Ciobanu, Dinu Zinovie, Dobi, Dorina, Liessi, Nara, Loberto, Nicoletta, Nobbio, Lucilla, Pedemonte, Nicoletta, Summa, Maria, Tomati, Valeria   +14 more
core   +1 more source

489 “Death doesn’t feel so immediate”: Changes in experiences of health before and after elexacaftor-tezacaftor-ivacaftor [PDF]

, 2023
Robin S. Everhart, E. McWilliams, K. Smith, K. Riekert   +3 more
openalex   +1 more source

Decline in HbA1c during the first year of elexacaftor/tezacaftor/ivacaftor treatment in the Danish cystic fibrosis cohort:Short title: Decline in HbA1c after elexacaftor/tezacaftor/ivacaftor treatment [PDF]

Background: Elexacaftor/tezacaftor/ivacaftor (ETI) has improved the clinical status of individuals with cystic fibrosis (CF), however, whether ETI impacts glucose tolerance remains unknown.
Almdal, Thomas Peter, Faurholt-Jepsen, Daniel, Jensen-Fangel, Søren, Katzenstein, Terese Lea, Mabuza Mathiesen, Inger Hee, Nielsen, Bibi Uhre, Olesen, Hanne Vebert, Olsen, Mette Frahm, Pressler, Tacjana, Ritz, Christian, Skov, Marianne   +10 more
core   +1 more source