Results 1 to 10 of about 5,337 (203)

Elexacaftor/Tezacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients With Cystic Fibrosis [PDF]

Clinical and Translational Science
Elexacaftor/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, Sihem Benaboud, Naïm Bouazza, Mahassen Barboura, Emmanuelle Bardin, Michel Miralles, Gabrielle Lui, Léo Froelicher‐Bournaud, Steeve Rouillon, Tiphaine Bihouee, Stéphanie Bui, Philippe Reix, Marie‐Laure Dalphin, Muriel Laurans, Jeanne Languepin, Harriet Corvol, Françoise Troussier, Laurence Weiss, Rames Cinthia, Aurélie Tatopoulos, Eric Deneuville, Raphael Chiron, Nathalie Stremler, Cathy Llerena, Sophie Ramel, Caroline Perisson, Véronique Houdoin, Marie Mittaine, Jean‐Marc Treluyer, Isabelle Sermet‐Gaudelus, Frantz Foissac, MODUL‐CF study group   +31 more
doaj   +3 more sources

Feasibility and Tolerance of High-Intensity Interval Training in Adults With Cystic Fibrosis [PDF]

Archives of Rehabilitation Research and Clinical Translation
Objectives: To assess the feasibility and the tolerance of a high-intensity interval training (HIIT) program compared with conventional rehabilitation during a 3-week hospital stay in adults with cystic fibrosis (CF) and to analyze the effectiveness and ...
Laurent Mely, PhD, Olivier Meurisse, PT, Laurene Norberciak, PhD, Morgane Auffret, CRNs, Nadège Goriot-Raynaud, NPs, Marion Buyse, PhD, Sophie Ramel, MD   +6 more
doaj   +2 more sources

Persistence of lung hyperinflation and small airway dysfunction in school-aged children with cystic fibrosis treated with elexacaftor–tezacaftor–ivacaftor: results from the real-world MODUL-CF study [PDF]

ERJ Open Research
Background Trapped gas (TG) and ventilation inhomogeneity (VI) are early markers of altered small airway function (SAF) in cystic fibrosis (CF) lung disease.
Lucie Colin, Véronique Delaup, Muriel Le Bourgeois, Véronique Neve, Nathalie Wizla-Derambure, Iulia-Cristina Ioan, Aurélie Tatopoulos, Sebastien Kieffer, Aurore Blonde, Plamen Bokov, Véronique Houdoin, Laurence Le Clainche, Audrey Barzic, Camille Ohlmann, Tom Toin, Thomas Perrin, Laurianne Coutier, Rola Abou Taam, Anne-Sophie Bonnel, Anthony Rignani, Hala Feghali, Stephanie Bui, Michael Fayon, Alexia Letierce, Isabelle Sermet-Gaudelus, Philippe Reix   +25 more
doaj   +2 more sources

Beyond Trikafta: new models to assess tissue dependent rescue of N1303K-CFTR [PDF]

Frontiers in Pharmacology
RationaleRespiratory 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, Iwona Pranke, Valeria Capurro, Benoit Chevalier, Benoit Chevalier, Emanuela Pesce, Valeria Tomati, Cristina Pastorino, Mairead Kelly-Aubert, Mairead Kelly-Aubert, Aurelie Hatton, Aurelie Hatton, Elise Dreano, Elise Dreano, Mariateresa Lena, Mariateresa Lena, Renata Bocciardi, Renata Bocciardi, Federico Zara, Federico Zara, Stefano Pantano, Vito Terlizzi, Cristina Lucanto, Stefano Costa, Laura Claut, Valeria Daccò, Piercarlo Poli, Massimo Maschio, Benedetta Fabrizzi, Benedetta Fabrizzi, Nicole Caporelli, Nicole Caporelli, Marco Cipolli, Sonia Volpi, Frederique Chedevergne, Laure Cosson, Julie Macey, Sophie Ramel, Laurence Weiss, Dominique Grenet, Laurence Le Clainche-Viala, Benoit Douvry, Bruno Ravoninjatovo, Camille Audousset, Aurélie Tatopoulos, Bénédicte Richaud-Thiriez, Melissa Baravalle, Guillaume Thouvenin, Guillaume Labbé, Marie Mittaine, Philippe Reix, Isabelle Durieu, Julie Mankikian, Stéphanie Bui, Thao Nguyen-Khoa, Thao Nguyen-Khoa, Thao Nguyen-Khoa, Karim Khoukh, Clémence Martin, Clémence Martin, Clémence Martin, Jennifer Da Silva, Jennifer Da Silva, Paola De Carli, Carlo Castellani, Federico Cresta, Luis Galietta, Anne Guillemaut, Emmanuelle Girodon, Emmanuelle Girodon, Emmanuelle Girodon, Natacha Remus, Mattijs Bulcaen, Marjolein Ensinck, Miroslaw Zajac, Marianne Carlon, Jean LeBihan, Pierre-Régis Burgel, Pierre-Régis Burgel, Pierre-Régis Burgel, Pierre-Régis Burgel, Isabelle Sermet-Gaudelus, Isabelle Sermet-Gaudelus, Isabelle Sermet-Gaudelus, Isabelle Sermet-Gaudelus, Alexandre Hinzpeter, Alexandre Hinzpeter, Nicoletta Pedemonte   +87 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]

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 ...
Pierre-Régis Burgel, Emmanuelle Girodon, Neeraj Sharma, Caroline Raynal, Jennifer Da Silva, Souphatta Sasorith, Clémence Martin, Isabelle Sermet-Gaudelus, Karen Raraigh   +8 more
doaj   +2 more sources

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, Hakim Rahmoune, Karim Bouziane-Nedjadi, Abdelkrim Radoui   +3 more
doaj   +1 more source

A conceptual framework to develop a patient-reported experience questionnaire on the cystic fibrosis journey in France: the ExPaParM collaborative study

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, A. Fanchini, P. Lombrail, G. Rault, A. Chansard, N. Le Breton, C. Frenod, F. Milon, C. Heymes Royer, D. Segretain, M. Silber, S. Therouanne, J. Haesebaert, C. Llerena, P. Michel, Q. Reynaud   +15 more
doaj   +1 more source

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, Albane Jouault, Alessandra Mattos Saliba, Lhousseine Touqui, Lhousseine Touqui   +4 more
doaj   +1 more source

Beneficial short-term effect of autogenic drainage on peripheral resistance in childhood cystic fibrosis disease

BMC Pulmonary Medicine, 2022
Background Airway clearance techniques are supposed to be a necessary adjunct for the enhancement of impaired peripheral clearance in cystic fibrosis (CF).
Plamen Bokov, Michèle Gerardin, Géraldine Brialix, Emmanuelle Da Costa Noble, Romain Juif, Antonia Vital Foucher, Laurence Le Clainche, Véronique Houdouin, Benjamin Mauroy, Christophe Delclaux   +9 more
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Estudo da freqüência de diabetes mellitus e intolerância à glicose em pacientes com fibrose cística Study of the frequency of diabetes mellitus and glucose intolerance in patients with cystic fibrosis

Jornal de Pediatria, 2001
OBJETIVO: avaliar a freqüência de diabetes mellitus (DM) e de intolerância à glicose nos pacientes com fibrose cística seguidos no Serviço de Gastropediatria do HC-FMRP-USP.
Flávia A.A. de Castro, Maria I.M. Fernandes, Renan M. Montenegro Júnior, Milton C. Foss   +3 more
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