Elexacaftor/Tezacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients With Cystic Fibrosis [PDF]
Clinical and Translational ScienceElexacaftor/tezacaftor/ivacaftor (ETI) significantly improves treatment outcomes for people with cystic fibrosis (pwCF) with at least one F508del allele. In 2023, the Food and Drug Administration approved ETI for children with CF aged 2–5 years. However,
Ngoc Hoa Truong +31 more
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Elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis harbouring two CFTR Class I variants: real-world data from the French compassionate programmeResearch in context [PDF]
EClinicalMedicineSummary: 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 ...
Pierre-Régis Burgel +8 more
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Beyond Trikafta: new models to assess tissue dependent rescue of N1303K-CFTR [PDF]
Frontiers in PharmacologyRationaleRespiratory status of people with Cystic Fibrosis (pwCF) carrying N1303K is improved by Elexacaftor/Tezacaftor/Ivacaftor (ETI) but, contrary to other mutations, the impact on sweat test results is limited.MethodsTo explore this discrepancy, we ...
Iwona Pranke +87 more
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Improvement of iron status with elexacaftor tezacaftor ivacaftor therapy is associated with the correction of systemic inflammation and improvement of lung function: a one-year prospective study [PDF]
Scientific ReportsIron deficiency (ID) is frequent in adult patients with cystic fibrosis (pwCF). The effect of elexacaftor-tezacaftor-ivacaftor (ETI) on iron metabolism has rarely been reported.
Hervé Lobbes +5 more
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Assessment of the efficacy of an antimicrobial peptide in the context of cystic fibrosis airways [PDF]
Current Research in Microbial SciencesAntimicrobial peptides (AMPs) offer a promising alternative to control airway infections with multi-resistant bacteria, such as methicillin-resistant Staphylococcus aureus (MRSA), which commonly infects patients with cystic fibrosis (CF).
Albane Jouault +3 more
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Manifestations digestives et hépatobiliaires des enfants atteints de mucoviscidose à Oran, Algérie [PDF]
Batna Journal of Medical Sciences, 2021 Introduction. La mucoviscidose reste une affection potentiellement sévère, responsable d’une morbimortalité élevée dans notre pays en raison du retard diagnostique et de prise en charge des enfants vu l’absence de dépistage néonatal.
Nada Boutrid +3 more
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Corrigendum: Long term NIV in an infant with Hallermann-Streiff syndrome: a case report and overview of respiratory morbidity [PDF]
Frontiers in PediatricsS. Guerin +4 more
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Orphanet Journal of Rare Diseases, 2023 Background The objective of the study was to elaborate a conceptual framework related to the domains of patient experience along the cystic fibrosis (CF) journey from the patients and parents of children with CF to inform the design of a patient-reported
D. Pougheon Bertrand +15 more
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Modulation of the immune response by the Pseudomonas aeruginosa type-III secretion system
Frontiers in Cellular and Infection Microbiology, 2022 Pseudomonas aeruginosa is an opportunistic pathogen that can cause critical cellular damage and subvert the immune response to promote its survival. Among the numerous virulence factors of P.
Albane Jouault +4 more
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