Evidence for Early Cystic Fibrosis Transmembrane Conductance Regulator Modulator Treatment for Children with Cystic Fibrosis Keeps Growing
- PMID: 35947636
- PMCID: PMC9746855
- DOI: 10.1164/rccm.202208-1507ED
Evidence for Early Cystic Fibrosis Transmembrane Conductance Regulator Modulator Treatment for Children with Cystic Fibrosis Keeps Growing
Comment on
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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 Study.Am J Respir Crit Care Med. 2022 Dec 1;206(11):1361-1369. doi: 10.1164/rccm.202202-0392OC. Am J Respir Crit Care Med. 2022. PMID: 35816621 Free PMC article. Clinical Trial.
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References
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- Despotes KA, Donaldson SH. Current state of CFTR modulators for treatment of cystic fibrosis. Curr Opin Pharmacol . 2022;65:102239. - PubMed
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- Cystic Fibrosis Foundation patient registry: 2020 Patient Registry Annual Data Report. Bethesda, MD: Cystic Fibrosis Foundation; 2021. https://www.cff.org/medical-professionals/patient-registry
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- Mall MA, Brugha R, Gartner S, Legg J, Moeller A, Mondejar-Lopez P, et al. VX19-445-116 Study Group 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 study. Am J Respir Crit Care Med . 2022;206:1361–1369. - PMC - PubMed
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