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Editorial
. 2022 Dec 1;206(11):1308-1310.
doi: 10.1164/rccm.202208-1507ED.

Evidence for Early Cystic Fibrosis Transmembrane Conductance Regulator Modulator Treatment for Children with Cystic Fibrosis Keeps Growing

Jordana E Hoppe  1   2 , Edith T Zemanick  1   2 , Stacey L Martiniano  1   2
Affiliations
Editorial

Evidence for Early Cystic Fibrosis Transmembrane Conductance Regulator Modulator Treatment for Children with Cystic Fibrosis Keeps Growing

Jordana E Hoppe et al. Am J Respir Crit Care Med. .
No abstract available

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Comment on

References

    1. Despotes KA, Donaldson SH. Current state of CFTR modulators for treatment of cystic fibrosis. Curr Opin Pharmacol . 2022;65:102239. - PubMed
    1. 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
    1. Zemanick ET, Taylor-Cousar JL, Davies J, Gibson RL, Mall MA, McKone EF, et al. 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 allele. Am J Respir Crit Care Med . 2021;203:1522–1532. - PMC - PubMed
    1. 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
    1. Middleton PG, Mall MA, Dřevínek P, Lands LC, McKone EF, Polineni D, et al. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del mutation. N Engl J Med . 2019;381:1809–1819. - PMC - PubMed