Results 21 to 30 of about 4,690 (200)

Elexacaftor/tezacaftor/ivacaftor projected survival and long-term health outcomes in people with cystic fibrosis homozygous for F508del.

Journal of Cystic Fibrosis, 2023
BACKGROUND A series of phase 3 clinical trials have demonstrated that elexacaftor plus tezacaftor plus ivacaftor (ELX/TEZ/IVA) is safe and efficacious in people with cystic fibrosis (pwCF) aged ≥12 years with ≥1 F508del mutation in the cystic fibrosis ...
A. Lopez, Conor Daly, G. Vega-Hernandez, G. MacGregor, J. Rubin   +4 more
semanticscholar   +1 more source

Individualized approach to elexacaftor/tezacaftor/ivacaftor dosing in cystic fibrosis, in response to self-reported anxiety and neurocognitive adverse events: A case series

Frontiers in Pharmacology, 2023
The prevalence of mental health disorders is high among people with Cystic Fibrosis. The psychological symptoms in CF are associated with poor adherence, worse treatment outcomes, and greater health utilization/cost.
Hisham Ibrahim, Hisham Ibrahim, Hammad Danish, David Morrissey, David Morrissey, Kevin F. Deasy, Kevin F. Deasy, Mairead McCarthy, James Dorgan, Claire Fleming, Ciara Howlett, Sarah Twohig, Tamara Vagg, Tamara Vagg, Desmond M. Murphy, Desmond M. Murphy, Michael Maher, Michael Maher, Barry J. Plant, Barry J. Plant   +19 more
doaj   +1 more source

Malassezia Folliculitis following Triple Therapy for Cystic Fibrosis

Medicina, 2022
Triple-combination therapy with elexacaftor, tezacaftor and ivacaftor has been recently approved for cystic fibrosis patients with at least one F508del mutation in the transmembrane conductance regulator of the cystic fibrosis gene.
Federica Li Pomi, Luca Di Bartolomeo, Mario Vaccaro, Maria Lentini, Simona Cristadoro, Maria Cristina Lucanto, Mariangela Lombardo, Stefano Costa, Francesco Borgia   +8 more
doaj   +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

Elexacaftor/tezacaftor/ivacaftor and gastrointestinal outcomes in cystic fibrosis: Report of promise-GI.

Journal of Cystic Fibrosis, 2022
BACKGROUND Elexacaftor/tezacaftor/ivacaftor (ETI) improves pulmonary disease in people with cystic fibrosis (PwCF), but its effect on gastrointestinal symptoms, which also affect quality of life, is not clear. METHODS PROMISE is a 56-center prospective,
S. Schwarzenberg, Phuong T. Vu, M. Skalland, L. Hoffman, C. Pope, D. Gelfond, M. Narkewicz, D. Nichols, S. Heltshe, S. Donaldson, C. Frederick, A. Kelly, J. Pittman, F. Ratjen, M. Rosenfeld, S. Sagel, G. Solomon, M. Stalvey, J. Clancy, S. Rowe, S. Freedman   +20 more
semanticscholar   +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

Impact of Pharmacy Services on Time to Elexacaftor-Tezacaftor-Ivacaftor Initiation [PDF]

Journal of Managed Care & Specialty Pharmacy, 2021
The approval of elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA) expanded highly effective cystic fibrosis transmembrane receptor modulator therapy to approximately 90% of persons age 12 and older with cystic fibrosis. Clinical pharmacists and pharmacy technicians played a key role in planning for ELX/TEZ/IVA initiation prior to FDA approval as well as ...
Lauren Roder, Michelle Simonsen, Lindsey Fitzpatrick, Jennifer Loucks, Jianghua He   +4 more
openaire   +2 more sources

Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study

American Journal of Respiratory and Critical Care Medicine, 2022
Rationale The triple-combination regimen elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in children aged 6 through 11 years with cystic fibrosis and at least one F508del-CFTR allele in a phase 3, open-label, single ...
M. Mall, R. Brugha, S. Gartner, J. Legg, A. Moeller, P. Mondéjar-López, Dario Prais, T. Pressler, F. Ratjen, P. Reix, P. Robinson, H. Selvadurai, F. Stehling, N. Ahluwalia, E. Arteaga‐Solis, B. Bruinsma, Mark Jennings, S. Moskowitz, S. Noel, S. Tian, Tanya G Weinstock, Pan Wu, C. Wainwright, J. Davies   +23 more
semanticscholar   +1 more source

Elexacaftor/tezacaftor/ivacaftor corrects monocyte microbicidal deficiency in cystic fibrosis

European Respiratory Journal, 2022
BackgroundCystic fibrosis (CF), which is caused by mutations in the CF transmembrane conductance regulator (CFTR), is characterised by chronic bacterial lung infection and inflammation. In CF, monocytes and monocyte-derived macrophages have been shown to display defective phagocytosis and antimicrobial activity against relevant lung pathogens ...
Luca Cavinato, Francesco R. Luly, Valentina Pastore, Daniele Chiappetta, Gloria Sangiorgi, Eva Ferrara, Pia Baiocchi, Giuseppe Mandarello, Giuseppe Cimino, Paola Del Porto, Fiorentina Ascenzioni   +10 more
openaire   +2 more sources