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The Lancet. Respiratory Medicine|February 13, 2021
Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension studyPatrick A Flume, Reta Fischer Biner, Damian G Downey, et al.
Annals of the American Thoracic Society|September 8, 2025
Evaluating Long-Term Effectiveness of Cystic Fibrosis Modulator Therapies After Rapid Adoption: A Dual-Approach StudyPedro Miranda Afonso, Grace C Zhou, Weiji Su, et al.
Pediatric Pulmonology|September 10, 2008
Safety of bronchoalveolar lavage in young children with cystic fibrosisClaire E Wainwright, Keith Grimwood, John B Carlin, et al.
American Journal of Respiratory and Critical Care Medicine|March 18, 2021
A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del AlleleEdith T Zemanick, Jennifer L Taylor-Cousar, Jane Davies, et al.
Pediatric Pulmonology|February 4, 2025
SHIFTing goals in cystic fibrosis-managing extrapulmonary disease in the era of CFTR modulator therapy; Proceedings of the International Shaping Initiatives and Future Trends (SHIFT) SymposiumJonathan E M O'Donnell, Lucy A Hastings, Julie N Briody, et al.
American Journal of Respiratory and Critical Care Medicine|July 11, 2022
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 StudyMarcus A Mall, Rossa Brugha, Silvia Gartner, et al.
Ebiomedicine|July 5, 2025
Phase 2a/b randomised placebo-controlled dose-escalation trial of triheptanoin for ataxia-telangiectasia: treating mitochondrial dysfunction with anaplerosisMatthew Lynch, Sophie Manoy, Peter D Sly, et al.
Nucleic Acids Research|November 30, 2013
PDBe: Protein Data Bank in EuropeAleksandras Gutmanas, Younes Alhroub, Gary M Battle, et al.
Nucleic Acids Research|October 18, 2015
PDBe: improved accessibility of macromolecular structure data from PDB and EMDBSameer Velankar, Glen van Ginkel, Younes Alhroub, et al.
Science (New York, N.Y.)|November 16, 2016
Emergence and spread of a human-transmissible multidrug-resistant nontuberculous mycobacteriumJosephine M Bryant, Dorothy M Grogono, Daniela Rodriguez-Rincon, et al.
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