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- Received December 20, 2022
- Accepted February 8, 2023
- Published online February 16, 2023.
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- Pierre-Régis Burgel1,2,3⇑,
- Isabelle Sermet-Gaudelus3,4,5,
- Isabelle Durieu3,6,7,
- Reem Kanaan2,3,
- Julie Macey8,
- Dominique Grenet9,
- Michele Porzio10,
- Nathalie Coolen-Allou11,
- Raphael Chiron12,
- Christophe Marguet13,
- Benoit Douvry14,
- Nadine Dufeu15,
- Isabelle Danner-Boucher16,
- Pierre Foucaud17,
- Lydie Lemonnier17,
- Emmanuelle Girodon18,
- Jennifer Da Silva2,3 and
- Clémence Martin1,2,3
- on Behalf of the French CF Reference Network study group
- 1Université Paris-Cité, Institut Cochin, Inserm U1016, Paris, France
- 2Respiratory Medicine and Cystic Fibrosis National Reference Center; Cochin Hospital; Assistance Publique Hôpitaux de Paris (AP-HP), Paris, France
- 3ERN-Lung CF network, Frankfurt, Germany
- 4Centre 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
- 5Université Paris-Cité, Institut Necker Enfants Malades, INSERM U1151, Paris, France
- 6Centre de référence Adulte de la Mucoviscidose, Service de médecine interne, Hospices civils de Lyon, Pierre Bénite, France
- 7Université de Lyon, Équipe d'Accueil Health Services and Performance Research (HESPER) 7425, Lyon, France
- 8Respiratory Medicine and Cystic Fibrosis Center, CHU de Bordeaux, Bordeaux, France
- 9CRCM - Centre de Transplantation Pulmonaire. Service de pneumologie, hôpital Foch, Suresnes, France
- 10Department of Respiratory Medicine and Cystic Fibrosis Center, Federation of Translational Medicine of Strasbourg (FMTS), University Hospitals, Strasbourg, France
- 11Centre Hospitalier Universitaire Félix Guyon, Saint Denis, La Réunion, France
- 12Cystic Fibrosis Center, Hôpital Arnaud de Villeneuve, Centre Hospitalier Universitaire de Montpellier, Montpellier, France
- 13Pediatric Respiratory Disease and Cystic Fibrosis Center, Hospital, UNIROUEN, Inserm EA 2656, Rouen University Hospital, Normandie Univ, Rouen, France
- 14Service de Pneumologie, Centre Hospitalier Intercommunal, FHU SENEC, Créteil, France
- 15Department of Respiratory Medicine and Lung Transplantation, Aix Marseille Univ, APHM, Hôpital Nord, Marseille, France
- 16Service de Pneumologie, L'Institut Du Thorax, CHU Nantes, Nantes, France
- 17Association Vaincre la Mucoviscidose, Paris, France
- 18APHP.Centre-Université de Paris Cité, Service de Médecine Génomique des Maladies de Système et d'Organe, Hôpital Cochin, Paris, France
- Corresponding author: Pierre Regis Burgel (pierre-regis.burgel{at}aphp.fr)
Funding
Association Vaincre la Mucoviscidose
Filière Muco CFTR
Société Française de la Mucoviscidose
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Vol 62 Issue 3
Table of Contents
The French Compassionate Program of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del CFTR variant
