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The expanded French compassionate programme for elexacaftor–tezacaftor–ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study

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Burgel, Pierre-Régis | Sermet-Gaudelus, Isabelle | Girodon, Emmanuelle | Durieu, Isabelle | Houdouin, Véronique | Audousset, Camille | Macey, Julie | Grenet, Dominique | Porzio, Michele | Murris-Espin, Marlène | Reix, Philippe | Baravalle, Mélisande | Belleguic, Chantal | Mely, Laurent | Verhille, Juliette | Weiss, Laurence | Reynaud-Gaubert, Martine | Mittaine, Marie | Hamidfar, Rebecca | Ramel, Sophie | Cosson, Laure | Douvry, Benoit | Danner-Boucher, Isabelle | Foucaud, Pierre | Roy, Charlotte | Burnet, Espérie | Raynal, Caroline | Audrezet, Marie-Pierre | da Silva, Jennifer | Martin, Clémence | Kanaan, Reem | Carlier, Nicolas | Honoré, Isabelle | Chedevergne, Frédérique | Dreano, Elise | Hatton, Aurélie | Hinzpeter, Alexandre | Pranke, Iwona | Le Clainche-Viala, Laurence | Mayer, Sophie | Corvol, Harriet | Thouvenin, Guillaume | de Miranda, Sandra | Remus, Natascha | Duthoit, Louise | Perez, Thierry | Le Rouzic, Olivier | Wizla, Nathalie | Bon, Claire | Bui, Stéphanie | Poey, Nora | Stremler, Nathalie | Coltey, Bérengère | Dufeu, Nadine | Lebihan, Jean | Gabsi, Asma | Pouradier, Delphine | Andrejak, Claire | Rames, Cinthia | Dupuy-Grasset, Magali | Languepin, Jeanne | Marguet, Christophe | Pramil, Stéphanie | Arnouat, Baptiste | Fanton, Annlyse | Abely, Michel | Ravoninjatovo, Bruno | Blondé, Aurore | Guillaumot, Anne | Kieffer, Sebastien | Tatopoulos, Aurélie | Nove-Josserand, Raphaële | Ohlmann, Camille | Perrin, Thomas | Reynaud, Quitterie | Llerena, Catherine | Quétant, Sébastien | Valois, Sophie | Dalphin, Marie-Laure | Richaud-Thiriez, Bénédicte | Deneuville, Eric | Chiron, Raphael | Socchi, Floriane | Bihouée, Tiphaine | Mankikian, Julie | Flament, Thomas | Coolen-Allou, Nathalie | Gachelin, Elsa | Périsson, Caroline | Vuillard, Constance | Dupuis, Marion | Alkoussa, Wael | Marchal, Sarah | Leroy, Sylvie | Scalbert, Manuela | Campbell, Karine | Laurans, Muriel | Labbé, Guillaume | Montcouquiol, Sylvie | Priou, Pascaline | de Carli, Paola | Lemonnier, Lydie | Dehillotte, Clémence | Nouvel, Thierry

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The expanded French compassionate programme for elexacaftor–tezacaftor–ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study

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International audience. Background Elexacaftor-tezacaftor-ivacaftor has been approved in Europe for people with cystic fibrosis with at least one F508del CFTR variant. Additionally, it is approved by the US Food and...

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Multisystemic Effects of Elexacaftor–Tezacaftor–Ivacaftor in Adults with Cystic Fibrosis and Advanced Lung Disease

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International audience. ationale: Limited data exist on the safety and effectiveness of elexacaftor-tezacaftor-ivacaftor (ETI) in people with cystic fibrosis (pwCF) and advanced lung disease. Objectives: To evaluate...

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