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Clinical and functional efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis carrying the N1303K mutation.
Sadras, Ido; Kerem, Eitan; Livnat, Galit; Sarouk, Ifat; Breuer, Oded; Reiter, Joel; Gileles-Hillel, Alex; Inbar, Ori; Cohen, Michael; Gamliel, Ayelet; Stanleigh, Noemie; Gunawardena, Tarini; Bartlett, Claire; Gonska, Tanja; Moraes, Theo; Eckford, Paul D W; Bear, Christine E; Ratjen, Felix; Kerem, Batsheva; Wilschanski, Michael; Shteinberg, Michal; Cohen-Cymberknoh, Malena.
  • Sadras I; Cystic Fibrosis Center, Hadassah Hebrew Medical Center and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Hebrew University of Jerusalem, Jerusalem 91120, Israel.
  • Kerem E; Cystic Fibrosis Center, Hadassah Hebrew Medical Center and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Hebrew University of Jerusalem, Jerusalem 91120, Israel. Electronic address: eitank@hadassah.org.il.
  • Livnat G; Cystic Fibrosis Center, Carmel Medical center, The Ruth and Bruce Rappaport Faculty of Medicine, Technion Israel Institute of Technology, Israel.
  • Sarouk I; Pediatric Pulmonology Unit, The National Center for Cystic Fibrosis, Sheba Medical Center, Tel Hashomer and the Sackler Faculty of Medicine, Safra Children's Hospital, Tel Aviv University, Israel.
  • Breuer O; Cystic Fibrosis Center, Hadassah Hebrew Medical Center and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Hebrew University of Jerusalem, Jerusalem 91120, Israel.
  • Reiter J; Cystic Fibrosis Center, Hadassah Hebrew Medical Center and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Hebrew University of Jerusalem, Jerusalem 91120, Israel.
  • Gileles-Hillel A; Cystic Fibrosis Center, Hadassah Hebrew Medical Center and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Hebrew University of Jerusalem, Jerusalem 91120, Israel.
  • Inbar O; The Cystic Fibrosis Foundation of Israel, Israel.
  • Cohen M; Cystic Fibrosis Center, Hadassah Hebrew Medical Center and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Hebrew University of Jerusalem, Jerusalem 91120, Israel.
  • Gamliel A; Cystic Fibrosis Center, Hadassah Hebrew Medical Center and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Hebrew University of Jerusalem, Jerusalem 91120, Israel.
  • Stanleigh N; Department of Genetics, The Life Sciences Institute, Hebrew University, Jerusalem Israel.
  • Gunawardena T; Molecular Medicine, Hospital for Sick Children, Toronto, ON, Canada; Translational Medicine, Hospital for Sick Children, Toronto, ON, Canada.
  • Bartlett C; Translational Medicine, Hospital for Sick Children, Toronto, ON, Canada.
  • Gonska T; Translational Medicine, Hospital for Sick Children, Toronto, ON, Canada; Division of Gastroenterology, Hepatology & Nutrition, Division of Respiratory Medicine, SickKids Hospital, Toronto, ON, Canada; Department of Physiology and Pediatrics, University of Toronto, Toronto, ON, Canada.
  • Moraes T; Translational Medicine, Hospital for Sick Children, Toronto, ON, Canada; Division of Gastroenterology, Hepatology & Nutrition, Division of Respiratory Medicine, SickKids Hospital, Toronto, ON, Canada; Department of Physiology and Pediatrics, University of Toronto, Toronto, ON, Canada.
  • Eckford PDW; Molecular Medicine, Hospital for Sick Children, Toronto, ON, Canada.
  • Bear CE; Molecular Medicine, Hospital for Sick Children, Toronto, ON, Canada; Department of Physiology and Pediatrics, University of Toronto, Toronto, ON, Canada.
  • Ratjen F; Translational Medicine, Hospital for Sick Children, Toronto, ON, Canada; Division of Gastroenterology, Hepatology & Nutrition, Division of Respiratory Medicine, SickKids Hospital, Toronto, ON, Canada; Department of Physiology and Pediatrics, University of Toronto, Toronto, ON, Canada.
  • Kerem B; Department of Genetics, The Life Sciences Institute, Hebrew University, Jerusalem Israel.
  • Wilschanski M; Cystic Fibrosis Center, Hadassah Hebrew Medical Center and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Hebrew University of Jerusalem, Jerusalem 91120, Israel.
  • Shteinberg M; Cystic Fibrosis Center, Carmel Medical center, The Ruth and Bruce Rappaport Faculty of Medicine, Technion Israel Institute of Technology, Israel.
  • Cohen-Cymberknoh M; Cystic Fibrosis Center, Hadassah Hebrew Medical Center and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Hebrew University of Jerusalem, Jerusalem 91120, Israel.
J Cyst Fibros ; 22(6): 1062-1069, 2023 Nov.
Article en En | MEDLINE | ID: mdl-37331863

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Fibrosis Quística Límite: Humans Idioma: En Año: 2023 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Fibrosis Quística Límite: Humans Idioma: En Año: 2023 Tipo del documento: Article