Results 161 to 170 of about 4,690 (200)

Elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis harbouring two CFTR Class I variants: real-world data from the French compassionate programme

EClinicalMedicine
Summary Background The European Medicines Agency has recently expanded the label of elexacaftor-tezacaftor-ivacaftor (ETI) to all people with cystic fibrosis (pwCF) aged 2 years and older who have at least one non-Class I mutation in the cystic fibrosis ...
Pierre-Régis Burgel, E. Girodon, N. Sharma, C. Raynal, J. da Silva, S. Sasorith, Clémence Martin, Isabelle Sermet-Gaudelus, K. Raraigh   +8 more
semanticscholar   +1 more source

Reported Adverse Events in Patients with CF Receiving Treatment with Elexacaftor/Tezacaftor/Ivacaftor: 5 Years Observational Study

Journal of Clinical Medicine
Background: Elexacaftor/tezacaftor/ivacaftor (ETI) treatment is showing remarkable beneficial effects in people with Cystic Fibrosis (pwCF) harboring the F508del mutation in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene.
Francesca Lucca, I. Meneghelli, G. Tridello, Francesca Buniotto, G. Cucchetto, Sonia Volpi, E. Pintani, V. Bezzerri, M. Cipolli   +8 more
semanticscholar   +1 more source

Effect of elexacaftor-tezacaftor-ivacaftor on liver transient elastography, fibrosis indices and blood tests in children with cystic fibrosis.

Journal of Cystic Fibrosis
BACKGROUND Elexacaftor-tezacaftor-ivacaftor (ETI) has significantly improved the clinical course of people with cystic fibrosis (pwCF) and eligible CFTR variants.
V. Terlizzi, C. Fevola, M. Cecchetti, A. Terminiello, F. Curci, E. Bartolini, C. Rubino, M. Stinco, S. Carrera, P. Bonomi, G. Taccetti, Zachary M. Sellers, G. Indolfi   +12 more
semanticscholar   +1 more source

Effects of elexacaftor–tezacaftor–ivacaftor discontinuation in cystic fibrosis

Respiratory Medicine and Research, 2022
Georgia Mitropoulou, Zisis Balmpouzis, Jérôme Plojoux, Jennifer Dotta-Celio, Alain Sauty, Angela Koutsokera   +5 more
openaire   +2 more sources

LONGITUDE: An observational study of the long-term effectiveness of elexacaftor/tezacaftor/ivacaftor in people aged ≥12 years with cystic fibrosis using data from the United Kingdom Cystic Fibrosis Registry - 2-year analysis.

Journal of Cystic Fibrosis
BACKGROUND The cystic fibrosis (CF) transmembrane conductance regulator modulator (CFTRm) elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has demonstrated efficacy and safety in clinical trials and emerging observational studies in people with CF.
G. Vega-Hernandez, G. MacGregor, A. Wilfin, F. Adams, C. Haugh, C. Baxter, H. Wöhling, Sarah L. Clarke, S. C. Charman, SB Carr   +9 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.G. 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

Heterogeneity in response to Elexacaftor/Tezacaftor/Ivacaftor in people with cystic fibrosis.

Journal of Cystic Fibrosis
BACKGROUND Highly effective modulators of the CFTR channel have been demonstrated to dramatically impact disease progression and outcome. However, real-world data indicates that the magnitude of the clinical benefit is not equal among all patients ...
G. Alicandro, A. Gramegna, F. Bellino, Sathya Calogero Sciarrabba, Chiara Lanfranchi, M. Contarini, M. Retucci, V. Daccò, Francesco Blasi   +8 more
semanticscholar   +1 more source

Outcomes of prenatal use of elexacaftor/tezacaftor/ivacaftor in carrier mothers to treat meconium ileus in fetuses with cystic fibrosis.

Journal of Cystic Fibrosis
As cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies including elexacaftor/tezacaftor/ivacaftor (ETI) have become widely used in eligible patients with cystic fibrosis (CF), the use of these medications in pregnant people has
A. Metcalf, S. Martiniano, S. Sagel, Michael V. Zaretsky, E. Zemanick, J. Hoppe   +5 more
semanticscholar   +1 more source

Fecal microbiota changes in people with cystic fibrosis after 6 months of elexacaftor/tezacaftor/ivacaftor: Findings from the promise study

Journal of Cystic Fibrosis
Background: People with cystic fibrosis (PwCF) often have fecal dysbioses relative to those without CF, characterized by increased pro-inflammatory microbiota and gastrointestinal (GI) inflammation as measured by fecal calprotectin, suggesting that ...
Jennifer T. Duong, Hillary S Hayden, A. Verster, Christopher Pope, Carson J. Miller, Kelsi Penewit, S. Salipante, Steven M Rowe, George M. Solomon, David Nichols, Andrea Kelly, S. Schwarzenberg, S.D. Freedman, Lucas R Hoffman   +13 more
semanticscholar   +1 more source

Peripheral Muscle Function and Body Composition in People With Cystic Fibrosis on Elexacaftor/Tezacaftor/Ivacaftor: A Cross‐Sectional Single‐Centre Study

Pediatric Pulmonology
People with cystic fibrosis (pwCF) often have multifactorial peripheral muscle abnormalities attributed to, for example, malnutrition, steroid use, altered redox balance and, potentially, CF‐specific intrinsic alterations. Malnutrition in CF now includes
L. Clayton, A. Shepherd, J. Corbett, M. Gruet, G. Connett, M. Allenby, Julian Legg, T. Daniels, D. S. Urquhart, Zoe L. Saynor   +9 more
semanticscholar   +1 more source