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Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rare CFTR variants: a viewpoint.
Burgel, Pierre-Régis; Sermet-Gaudelus, Isabelle; Girodon, Emmanuelle; Kanaan, Reem; Le Bihan, Jean; Remus, Natascha; Ravoninjatovo, Bruno; Grenet, Dominique; Porzio, Michele; Houdouin, Véronique; Le Clainche-Viala, Laurence; Durieu, Isabelle; Nove-Josserand, Raphaele; Languepin, Jeanne; Coltey, Bérangère; Guillaumot, Anne; Audousset, Camille; Chiron, Raphaël; Weiss, Laurence; Fajac, Isabelle; Da Silva, Jennifer; Martin, Clémence.
Afiliação
  • Burgel PR; Université Paris-Cité, Institut Cochin, Inserm U1016, Paris, France pierre-regis.burgel@aphp.fr.
  • Sermet-Gaudelus I; Respiratory Medicine and Cystic Fibrosis National Reference Center, Cochin Hospital, Assistance Publique Hôpitaux de Paris (AP-HP), Paris, France.
  • Girodon E; ERN-Lung CF network, Frankfurt, Germany.
  • Kanaan R; ERN-Lung CF network, Frankfurt, Germany.
  • Le Bihan J; 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.
  • Remus N; Université Paris-Cité, Institut Necker Enfants Malades, INSERM U1151, Paris, France.
  • Ravoninjatovo B; APHP.Centre-Université de Paris Cité, Service de Médecine Génomique des Maladies de Système et d'Organe, Hôpital Cochin, Paris, France.
  • Grenet D; Respiratory Medicine and Cystic Fibrosis National Reference Center, Cochin Hospital, Assistance Publique Hôpitaux de Paris (AP-HP), Paris, France.
  • Porzio M; ERN-Lung CF network, Frankfurt, Germany.
  • Houdouin V; CF Centre, Centre Perharidy, Service de Soins de Suite Nutritionnelle et Respiratoire, Roscoff, France.
  • Le Clainche-Viala L; Centre Hospitalier Intercommunal de Créteil, Service de Pédiatrie Générale, Créteil, France.
  • Durieu I; Department of Respiratory Diseases, Reims University Hospital, Reims, France.
  • Nove-Josserand R; CRCM - Centre de Transplantation Pulmonaire, Service de pneumologie, hôpital Foch, Suresnes, France.
  • Languepin J; Department of Respiratory Medicine and Cystic Fibrosis Center, Federation of Translational Medicine of Strasbourg (FMTS), University Hospitals, Strasbourg, France.
  • Coltey B; Université Paris-Cité, Institut Cochin, Inserm U1016, Paris, France.
  • Guillaumot A; Hôpital Robert Debré, Paris, France.
  • Audousset C; Hôpital Robert Debré, Paris, France.
  • Chiron R; ERN-Lung CF network, Frankfurt, Germany.
  • Weiss L; Centre de référence Adulte de la Mucoviscidose, Service de médecine interne, Hospices civils de Lyon, Pierre Bénite, France.
  • Fajac I; Université de Lyon, Research on Healthcare Performance (RESHAPE), INSERM U1290, Lyon, France.
  • Da Silva J; ERN-Lung CF network, Frankfurt, Germany.
  • Martin C; Centre de référence Adulte de la Mucoviscidose, Service de médecine interne, Hospices civils de Lyon, Pierre Bénite, France.
Eur Respir J ; 63(1)2024 Jan.
Article em En | MEDLINE | ID: mdl-38242629
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Pirazóis / Piridinas / Pirrolidinas / Quinolonas / Fibrose Cística / Benzodioxóis / Aminofenóis / Indóis Limite: Humans Idioma: En Revista: Eur Respir J Ano de publicação: 2024 Tipo de documento: Article País de afiliação: França
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Pirazóis / Piridinas / Pirrolidinas / Quinolonas / Fibrose Cística / Benzodioxóis / Aminofenóis / Indóis Limite: Humans Idioma: En Revista: Eur Respir J Ano de publicação: 2024 Tipo de documento: Article País de afiliação: França