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Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study.
Mall, Marcus A; Brugha, Rossa; Gartner, Silvia; Legg, Julian; Moeller, Alexander; Mondejar-Lopez, Pedro; Prais, Dario; Pressler, Tacjana; Ratjen, Felix; Reix, Philippe; Robinson, Paul D; Selvadurai, Hiran; Stehling, Florian; Ahluwalia, Neil; Arteaga-Solis, Emilio; Bruinsma, Bote G; Jennings, Mark; Moskowitz, Samuel M; Noel, Sabrina; Tian, Simon; Weinstock, Tanya G; Wu, Pan; Wainwright, Claire E; Davies, Jane C.
Afiliación
  • Mall MA; Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin and.
  • Brugha R; Berlin Institute of Health at Charité - Universitätsmedizin Berlin, Berlin, Germany.
  • Gartner S; German Center for Lung Research, Associated Partner, Berlin, Germany.
  • Legg J; Great Ormond Street Hospital for Children, London, United Kingdom.
  • Moeller A; Hospital Universitari Vall d'Hebron, Barcelona, Spain.
  • Mondejar-Lopez P; Southampton Children's Hospital, Hampshire, United Kingdom.
  • Prais D; University Children's Hospital, Zurich, Switzerland.
  • Pressler T; Hospital Clinico Universitario Virgen de la Arrixaca, Murcia, Spain.
  • Ratjen F; Schneider Children's Medical Center of Israel, Petah Tikva, Israel.
  • Reix P; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
  • Robinson PD; Copenhagen University Hospital, Rigshospitalet, Denmark.
  • Selvadurai H; The Hospital for Sick Children, Toronto, Ontario, Canada.
  • Stehling F; Hôpital Femme Mère-Enfant, Hospices Civils de Lyon, Bron, France.
  • Ahluwalia N; The Children's Hospital at Westmead, Sydney Children's Hospital Network, Sydney, Australia.
  • Arteaga-Solis E; The Children's Hospital at Westmead, Sydney Children's Hospital Network, Sydney, Australia.
  • Bruinsma BG; Universitätsklinikum Essen, Klinik für Kinderheilkunde III, Essen, Germany.
  • Jennings M; Vertex Pharmaceuticals Incorporated, Boston, Massachussetts.
  • Moskowitz SM; Vertex Pharmaceuticals Incorporated, Boston, Massachussetts.
  • Noel S; Vertex Pharmaceuticals Incorporated, Boston, Massachussetts.
  • Tian S; Vertex Pharmaceuticals Incorporated, Boston, Massachussetts.
  • Weinstock TG; Vertex Pharmaceuticals Incorporated, Boston, Massachussetts.
  • Wu P; Vertex Pharmaceuticals Incorporated, Boston, Massachussetts.
  • Wainwright CE; Vertex Pharmaceuticals Incorporated, Boston, Massachussetts.
  • Davies JC; Vertex Pharmaceuticals Incorporated, Boston, Massachussetts.
Am J Respir Crit Care Med ; 206(11): 1361-1369, 2022 12 01.
Article en En | MEDLINE | ID: mdl-35816621

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Regulador de Conductancia de Transmembrana de Fibrosis Quística / Fibrosis Quística Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Child / Humans Idioma: En Año: 2022 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Regulador de Conductancia de Transmembrana de Fibrosis Quística / Fibrosis Quística Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Child / Humans Idioma: En Año: 2022 Tipo del documento: Article