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Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study.
Chilvers, Mark A; Davies, Jane C; Milla, Carlos; Tian, Simon; Han, Zifei; Cornell, Alexandra G; Owen, Caroline A; Ratjen, Felix.
Affiliation
  • Chilvers MA; BC Children's Hospital, Vancouver, BC, Canada. Electronic address: mchilvers@cw.bc.ca.
  • Davies JC; National Heart and Lung Institute, Imperial College London, and Royal Brompton & Harefield NHS Foundation Trust, London, UK.
  • Milla C; Center for Excellence in Pulmonary Biology, Stanford University School of Medicine, Palo Alto, CA, USA.
  • Tian S; Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
  • Han Z; formerly of Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
  • Cornell AG; Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
  • Owen CA; Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
  • Ratjen F; The Hospital for Sick Children, Toronto, ON, Canada.
Lancet Respir Med ; 9(7): 721-732, 2021 07.
Article in En | MEDLINE | ID: mdl-33516285
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Quinolones / Cystic Fibrosis Transmembrane Conductance Regulator / Cystic Fibrosis / Benzodioxoles / Aminophenols / Aminopyridines / Mutation Type of study: Clinical_trials / Prognostic_studies Limits: Child / Female / Humans / Male Country/Region as subject: America do norte / Europa / Oceania Language: En Journal: Lancet Respir Med Year: 2021 Document type: Article Country of publication: United kingdom
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Quinolones / Cystic Fibrosis Transmembrane Conductance Regulator / Cystic Fibrosis / Benzodioxoles / Aminophenols / Aminopyridines / Mutation Type of study: Clinical_trials / Prognostic_studies Limits: Child / Female / Humans / Male Country/Region as subject: America do norte / Europa / Oceania Language: En Journal: Lancet Respir Med Year: 2021 Document type: Article Country of publication: United kingdom