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The New England Journal of Medicine, ISSN 0028-4793, 11/2011, Volume 365, Issue 18, pp. 1663 - 1672
Journal Article
2003, ISBN 0306478374, 228
Book
Journal Article
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 4/2012, Volume 109, Issue 15, pp. 5809 - 5814
Journal Article
Nature medicine, ISSN 1078-8956, 2010, Volume 16, Issue 9, pp. 1018 - 1023
Journal Article
European Respiratory Journal, ISSN 0903-1936, 10/2014, Volume 44, Issue 4, pp. 1042 - 1054
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 11/2017, Volume 377, Issue 21, pp. 2024 - 2035
Approximately 5% of patients with cystic fibrosis express one allele with some retained CFTR function. In a prospective trial, tezacaftor–ivacaftor had a... 
POPULATION | REGULATOR | MEDICINE, GENERAL & INTERNAL | LUMACAFTOR | EFFICACY | SAFETY | PHENOTYPE | AGED 6-11 YEARS | RANDOMIZED CONTROLLED-TRIAL | F508DEL-CFTR | G551D-CFTR MUTATION | Forced Expiratory Volume - drug effects | Cystic Fibrosis - physiopathology | Humans | Male | Aminophenols - therapeutic use | Quinolones - pharmacology | Aminophenols - adverse effects | Aminophenols - pharmacology | Young Adult | Quinolones - adverse effects | Quinolones - therapeutic use | Cystic Fibrosis Transmembrane Conductance Regulator - pharmacology | Adult | Benzodioxoles - adverse effects | Female | Indoles - pharmacology | Benzodioxoles - therapeutic use | Child | Double-Blind Method | Cross-Over Studies | Cystic Fibrosis Transmembrane Conductance Regulator - therapeutic use | Indoles - adverse effects | Cystic Fibrosis - genetics | Cystic Fibrosis Transmembrane Conductance Regulator - adverse effects | Adolescent | Quality of Life | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Heterozygote | Indoles - therapeutic use | Mutation | Benzodioxoles - pharmacology | Cystic Fibrosis - drug therapy | Drug Combinations | Proteins | Clonal deletion | Clinical trials | Heterozygotes | Cystic fibrosis | Cystic fibrosis transmembrane conductance regulator | Gene deletion | Drug therapy | Patients | Pharmaceuticals | Index Medicus | Abridged Index Medicus
Journal Article
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 07/2014, Volume 124, Issue 7, pp. 3047 - 3060
Journal Article