The French compassionate programme of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del CFTR variant.

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Title: The French compassionate programme of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del CFTR variant.
Authors: Burgel PR; Université Paris-Cité, Institut Cochin, Inserm U1016, Paris, France pierre-regis.burgel@aphp.fr.; Respiratory Medicine and Cystic Fibrosis National Reference Centre, Cochin Hospital, Assistance Publique Hôpitaux de Paris (AP-HP), Paris, France.; ERN-Lung CF Network, Frankfurt, Germany., Sermet-Gaudelus I; ERN-Lung CF Network, Frankfurt, Germany.; Centre de de Référence Maladies Rares, Mucoviscidose et affections liées à CFTR, Pneumologie Pédiatrique et Allergologie, Hôpital Necker Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.; Université Paris-Cité, Institut Necker Enfants Malades, INSERM U1151, Paris, France., Durieu I; ERN-Lung CF Network, Frankfurt, Germany.; Centre de Référence Adulte de la Mucoviscidose, Service de Médecine Interne, Hospices Civils de Lyon, Pierre Bénite, France.; Université de Lyon, Équipe d'Accueil Health Services and Performance Research (HESPER) 7425, Lyon, France., Kanaan R; Respiratory Medicine and Cystic Fibrosis National Reference Centre, Cochin Hospital, Assistance Publique Hôpitaux de Paris (AP-HP), Paris, France.; ERN-Lung CF Network, Frankfurt, Germany., Macey J; Respiratory Medicine and Cystic Fibrosis Center, CHU de Bordeaux, Bordeaux, France., Grenet D; CRCM -‌ Centre de Transplantation Pulmonaire, Service de Pneumologie, Hôpital Foch, Suresnes, France., Porzio M; Department of Respiratory Medicine and Cystic Fibrosis Centre, Federation of Translational Medicine of Strasbourg (FMTS), University Hospitals, Strasbourg, France., Coolen-Allou N; Centre Hospitalier Universitaire Félix Guyon, Saint Denis, La Réunion, France., Chiron R; Cystic Fibrosis Center, Hôpital Arnaud de Villeneuve, Centre Hospitalier Universitaire de Montpellier, Montpellier, France., Marguet C; Pediatric Respiratory Disease and Cystic Fibrosis Centre, Hospital, UNIROUEN, Inserm EA 2656, Rouen University Hospital, Normandie Univ, Rouen, France., Douvry B; Service de Pneumologie, Centre Hospitalier Intercommunal, FHU SENEC, Créteil, France., Dufeu N; Department of Respiratory Medicine and Lung Transplantation, Aix Marseille Univ, APHM, Hôpital Nord, Marseille, France., Danner-Boucher I; Service de Pneumologie, L'Institut Du Thorax, CHU Nantes, Nantes, France., Foucaud P; Association Vaincre la Mucoviscidose, Paris, France., Lemonnier L; Association Vaincre la Mucoviscidose, Paris, France., Girodon E; APHP Centre-Université de Paris Cité, Service de Médecine Génomique des Maladies de Système et d'Organe, Hôpital Cochin, Paris, France., Da Silva J; Respiratory Medicine and Cystic Fibrosis National Reference Centre, Cochin Hospital, Assistance Publique Hôpitaux de Paris (AP-HP), Paris, France.; ERN-Lung CF Network, Frankfurt, Germany., Martin C; Université Paris-Cité, Institut Cochin, Inserm U1016, Paris, France.; Respiratory Medicine and Cystic Fibrosis National Reference Centre, Cochin Hospital, Assistance Publique Hôpitaux de Paris (AP-HP), Paris, France.; ERN-Lung CF Network, Frankfurt, Germany., Andrejak C, Priou P, Richaud-Thiriez B, Macey J, Montcouquiol S, Douvry B, Remus N, Fanton A, Coolen-Allou N, Gachelin E, Vuillard C, Audousset C, Duthoit L, Bellet-Fraysse E, Languepin J, Durieu I, Nove-Josserand R, Ohlmann C, Raynaud Q, Dufeu N, Chiron R, Billon Y, Danner-Boucher I, Tissot A, Leroy S, Aubourg F, Burnet E, Burgel PR, Carlier N, Da Silva J, Fajac I, Girodon E, Kanaan R, Honoré I, Martin C, Le Bourgeois M, Sermet-Gaudelus I, Le Clainche-Viala L, Corvol H, Dehillotte C, Foucaud P, Lemonnier L, Brinchault G, Ravoninjatovo B, Le Bihan J, Ramel S, Marguet C, Porzio M, Weiss L, Grenet D, de Miranda S, Cosson L, Mankikian J, Pouradier D
Corporate Authors: French CF Reference Network study group
Source: The European respiratory journal [Eur Respir J] 2023 May 05; Vol. 61 (5). Date of Electronic Publication: 2023 May 05 (Print Publication: 2023).
Publication Type: Journal Article
Journal Info: Publisher: European Respiratory Society Country of Publication: England NLM ID: 8803460 Publication Model: Electronic-Print Cited Medium: Internet ISSN: 1399-3003 (Electronic) Linking ISSN: 09031936 NLM ISO Abbreviation: Eur Respir J Subsets: MEDLINE; PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1399-3003
DOI:10.1183/13993003.02437-2022