Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study.

Davies, Jane C; Cunningham, Steve; Harris, William T; Lapey, Allen; Regelmann, Warren E; Sawicki, Gregory S; Southern, Kevin W; Robertson, Sarah; Green, Yulia; Cooke, Jon; Rosenfeld, Margaret

Davies, Jane C; Cunningham, Steve; Harris, William T; Lapey, Allen; Regelmann, Warren E; Sawicki, Gregory S; Southern, Kevin W; Robertson, Sarah; Green, Yulia; Cooke, Jon; Rosenfeld, Margaret.

Affiliation

Davies JC; Imperial College London, London, UK; Royal Brompton and Harefield NHS Foundation Trust, London, UK. Electronic address: j.c.davies@imperial.ac.uk.
Cunningham S; Royal Hospital for Sick Children, NHS Lothian, Edinburgh, UK.
Harris WT; University of Alabama at Birmingham, Birmingham, AL, USA.
Lapey A; Massachusetts General Hospital, Boston, MA, USA.
Regelmann WE; University of Minnesota, Minneapolis, MN, USA.
Sawicki GS; Harvard Medical School, Boston, MA, USA.
Southern KW; University of Liverpool, Liverpool, UK.
Robertson S; Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
Green Y; Vertex Pharmaceuticals Incorporated, Milton Park, UK.
Cooke J; Vertex Pharmaceuticals Incorporated, Milton Park, UK.
Rosenfeld M; Seattle Children's Hospital, Seattle, WA, USA.

Lancet Respir Med ; 4(2): 107-15, 2016 Feb.

Article de En | MEDLINE | ID: mdl-26803277

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Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Sueur / Chlorures / Quinolinone / Protéine CFTR / Mucoviscidose / Agonistes de canaux chlorure / Aminophénols Type d'étude: Clinical_trials Limites: Child, preschool / Female / Humans / Male Langue: En Journal: Lancet Respir Med Année: 2016 Type de document: Article

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