Results 31 to 40 of about 3,103 (172)

Outcome of elexacaftor/tezacaftor/ivacaftor therapy in patients with cystic fibrosis and solid organ transplantation

, 2023
We report a case series of four patients with cystic fibrosis (CF) and previous solid organ transplantation (SOT) receiving elexacaftor/tezacaftor/ivacaftor therapy for 6 months or more. Data was collected retrospectively.
Peter Ott, Qvist, Tavs, Majbritt Jeppesen, Terese L. Katzenstein, Frederikke F. Rönsholt, Tavs Qvist, Ørum, Matilde B., Bendstrup, Elisabeth, Perch, Michael, Michael Perch, Pressler, Tacjana, Elisabeth Bendstrup, Jensen-Fangel, Søren, Ott, Peter, Katzenstein, Terese L., Tacjana Pressler, Rönsholt, Frederikke F., Matilde B. Ørum, Jeppesen, Majbritt, Søren Jensen‐Fangel   +19 more
core   +1 more source

Lingering Identity as Chronically Ill and the Unanticipated Effects of Life-Changing Precision Medicine in Cystic Fibrosis: A Case Report

Journal of Patient Experience, 2021
Cystic fibrosis (CF) is the leading genetic disease among Caucasians; however, advances in diagnosis and treatment have improved both quality and quantity of life for those affected.
Sigrid Ladores PhD, RN, PNP, CNE, FAAN, Morgan Polen   +1 more
doaj   +1 more source

Aplasia Cutis Congenita in the Setting of Maternal Cystic Fibrosis

Annals of Internal Medicine: Clinical Cases, 2022
Aplasia cutis congenita is a rare scalp defect. An affected neonate was delivered after in utero exposure beginning in the midtrimester to elexacaftor/tezacaftor/ivacaftor for maternal cystic fibrosis management. Although aplasia cutis congenita has many
Govind Kallumkal, Robert Egerman
doaj   +1 more source

Rescue by elexacaftor-tezacaftor-ivacaftor of the G1244E cystic fibrosis mutation's stability and gating defects are dependent on cell background

, 2023
Background: Cystic fibrosis is caused by mutations impairing expression, trafficking, stability and/or activity of the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel.
Di Duca, Marco, Lena, Mariateresa, Cresta, Federico, Galietta, Luis Juan Vicente, Bocciardi, Renata, Pastorino, Cristina, Costa, Stefano, Tomati, Valeria, Pesce, Emanuela, Castellani, Carlo, Capurro, Valeria, Galietta, Luis J V, Cristadoro, Simona, Zara, Federico, Pedemonte, Nicoletta, Lucanto, Maria Cristina, Sondo, Elvira   +16 more
core   +1 more source

Physiologically Based Pharmacokinetic Modeling of CFTR Modulation in People with Cystic Fibrosis Transitioning from Mono or Dual Regimens to Triple-Combination Elexacaftor/Tezacaftor/Ivacaftor

Pulmonary Therapy, 2020
Introduction The triple-combination (TC) cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen elexacaftor, tezacaftor, and ivacaftor was shown to be safe and efficacious in phase 3 trials of people with cystic fibrosis (pwCF) ≥ 12 
Alice Tsai, Shu-Pei Wu, Eric Haseltine, Sanjeev Kumar, Samuel M. Moskowitz, Paul Panorchan, Kushal Shah   +6 more
doaj   +1 more source

Novel reaction to new cystic fibrosis medication Trikafta

Clinical Case Reports, 2021
We present a novel case of an urticaria multiforme‐type drug reaction to the new cystic fibrosis medication Trikafta (elexacaftor + tezacaftor + ivacaftor).
Julian Stashower, Patrick Carr, Virginia Miller, Barrett Zlotoff   +3 more
doaj   +1 more source

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

Elexacaftor/Ivacaftor/Tezacaftor: First Approval

, 2019
Compliance with Ethical StandardsFunding: The preparation of this review was not supported by any external funding. Conflict of interest: Sheridan Hoy is a salaried employee of Adis International Ltd/Springer Nature, is responsible for the article ...
Sheridan M. Hoy (4897468)
core   +1 more source

Targeting the E1 ubiquitin-activating enzyme (UBA1) improves elexacaftor/tezacaftor/ivacaftor efficacy towards F508del and rare misfolded CFTR mutants [PDF]

, 2022
The advent of Trikafta (Kaftrio in Europe) (a triple-combination therapy based on two correctors-elexacaftor/tezacaftor-and the potentiator ivacaftor) has represented a revolution for the treatment of patients with cystic fibrosis (CF) carrying the most ...
D’Amore, Claudio, Cresta, Federico, Salvi, Mauro, Tomati, Valeria, Pedemonte, Nicoletta, Borgo, Christian, Castellani, Carlo, Capurro, Valeria, Sondo, Elvira   +8 more
core   +1 more source

Combined CFTR modulator therapies are linked with anabolic benefits and insulin-sparing in cystic fibrosis-related diabetes

Journal of Clinical & Translational Endocrinology, 2023
Aims: Combined CFTR modulator therapies have dramatically altered pulmonary outcomes in patients with cystic fibrosis (CF). Their impact on glucose metabolism requires further investigations.
Fabian Lurquin, Sophie Gohy, Michel P. Hermans, Vanessa Preumont   +3 more
doaj   +1 more source