Results 21 to 30 of about 9,288 (216)

Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience

Pulmonary Therapy, 2020
Plain Language Summary We performed a study to better understand the long-term impact of treatment with a drug called ivacaftor for patients with cystic fibrosis (CF).
Mark Higgins, Nataliya Volkova, Kristin Moy, Bruce C. Marshall, Diana Bilton   +4 more
doaj   +1 more source

Frequency of CFTR variants in southern Brazil and indication for modulators therapy in patients with cystic fibrosis

Genetics and Molecular Biology, 2021
This is a descriptive cross-sectional study that aims to determine the distribution of the CFTR causing variant in a group of patients at a cystic fibrosis (CF) center in southern Brazil, as well as to describe causing variants that are treatable with ...
Eliandra da Silveira Lima, Luíse Sgarabotto Pezzin, Ana Carolina Fensterseifer, Leonardo Araújo Pinto   +3 more
doaj   +1 more source

Elexacaftor-Tezacaftor-Ivacaftor and Ivacaftor (Trikafta)

Canadian Journal of Health Technologies, 2021
CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments ...
openaire   +3 more sources

Long-Term Ivacaftor in People Aged 6 Years and Older with Cystic Fibrosis with Ivacaftor-Responsive Mutations

Pulmonary Therapy, 2020
Introduction Mutations in the cystic fibrosis transmembrane conductance regulator gene (CFTR) affect the quantity and/or function of CFTR protein reaching the cell surface.
Joseph M. Pilewski, Kris De Boeck, Jerry A. Nick, Simon Tian, Cynthia DeSouza, Mark Higgins, Richard B. Moss   +6 more
doaj   +1 more source

Ivacaftor Inhibits Glioblastoma Stem Cell Maintenance and Tumor Progression

Frontiers in Cell and Developmental Biology, 2021
Glioblastoma (GBM) is the most common and malignant primary brain tumor. Glioblastoma stem cells (GSCs) not only initiate and sustain uncontrolled cell proliferation but also resistant to conventional clinical therapies including temozolomide (TMZ ...
Kun Liu, Kun Liu, Jun Pu, Zhi Nie, Zhi Nie, Yulin Shi, Yulin Shi, Liping Jiang, Qisheng Wu, Yongbin Chen, Yongbin Chen, Yongbin Chen, Cuiping Yang, Cuiping Yang   +13 more
doaj   +1 more source

Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2–5 Years with Cystic Fibrosis and at Least One F508del Allele

American Journal of Respiratory and Critical Care Medicine, 2023
Rationale Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and effective in people with cystic fibrosis (CF) aged ⩾6 years with at least one F508del-CFTR allele but has not been studied in younger children.
J. Goralski, J. Hoppe, M. Mall, S. McColley, E. McKone, B. Ramsey, J. Rayment, P. Robinson, F. Stehling, J. Taylor-Cousar, E. Tullis, N. Ahluwalia, A. Chin, C. Chu, Mengdi Lu, Tao Niu, Tanya G. Weinstock, F. Ratjen, M. Rosenfeld   +18 more
semanticscholar   +1 more source

Adherence to Ivacaftor is suboptimal [PDF]

Journal of Cystic Fibrosis, 2015
Ivacaftor, the first cystic fibrosis (CF) transmembrane conductance regulator (CFTR) potentiator provides a remarkable example of personalised medicine that has the capacity to transform lives and patient care. For those with a CFTR-G551D mutation, two phase three trials (adult and paediatric age 6+ years) and a follow-up open label trial have ...
Abbott, Janice, Bilton, Diana
openaire   +2 more sources

Real-world safety and effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: Interim results of a long-term registry-based study.

Journal of Cystic Fibrosis, 2023
BACKGROUND Phase 3 clinical trials showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in people with cystic fibrosis (CF) with ≥1 F508del-CFTR allele.
J. Bower, N. Volkova, N. Ahluwalia, G. Sahota, F. Xuan, A. Chin, Tanya G. Weinstock, Josh Ostrenga, A. Elbert   +8 more
semanticscholar   +1 more source

Global prevalence of CFTR variants with respect to their responsiveness to elexacaftor-tezacaftor-ivacaftor.

Journal of Cystic Fibrosis
BACKGROUND Elexacaftor-tezacaftor-ivacaftor (ETI) is mostly approved in people with cystic fibrosis (pwCF) with a p.Phe508del CFTR variant. The US Food and Drug Administration (FDA) approved ETI for an additional 177 rare CFTR variants and studies ...
P. Burgel, A. Orenti, E. Cromwell, M. Macek, H. Gutierrez, B. Karadag, A. Faro, J. V. van Rens, L. Naehrlich, E. Bakkeheim, S. Carr, A. Lindblad, A. Zolin, E. Lammertyn, R. Ruseckaite, M. Zampoli, C. Byrnes, Lvrf da Silva-Filho, A. Elbert, S.Y. Cheng, A. Stephenson   +20 more
semanticscholar   +1 more source

The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients.

PLoS ONE, 2020
ImportanceSinonasal symptoms in patients suffering from cystic fibrosis can negatively influence the quality of life and sinuses can be a niche for pathogens causing infection and inflammation leading to a decrease of lung function.
Romee Gostelie, Inge Stegeman, Gitte Berkers, Joost Bittermann, Ivonne Ligtenberg-van der Drift, Peter-Jan van Kipshagen, Karin de Winter-de Groot, Lucienne Speleman   +7 more
doaj   +1 more source