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Editorial
. 2021 Jun 15;203(12):1453-1455.
doi: 10.1164/rccm.202104-0850ED.

The Future of Highly Effective Modulator Therapy in Cystic Fibrosis

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Editorial

The Future of Highly Effective Modulator Therapy in Cystic Fibrosis

Cori L Daines et al. Am J Respir Crit Care Med. .
No abstract available

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Figures

Figure 1.
Figure 1.
Schematic representation of two potential treatment responses. Treatment A acutely improves FEV1 but does not slow rate of decline in FEV1, resulting in a minor improvement in the long-term outcome. Treatment B does not acutely improve FEV1 but does slow the rate of decline in FEV1, resulting in sustained benefit.

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References

    1. Ranganathan SC, Hall GL, Sly PD, Stick SM, Douglas TA. AREST-CF. Early lung disease in infants and preschool children with cystic fibrosis. Am J Respir Crit Care Med. 2017;195:1567–1575. - PMC - PubMed
    1. Sanders DB, Fink A, Mayer-Hamblett N, Schechter MS, Sawicki GS, Rosenfeld M, et al. Early life growth trajectories in cystic fibrosis are associated with pulmonary function at age 6 years. J Pediatr. 2015;167:1081–8.e1. - PMC - PubMed
    1. Zemanick ET, Taylor-Cousar JL, Davies J, Gibson RL, Mall MA, McKone EF, et al. VX18-445-106 Study Group. 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. Heijerman HGM, KcKone EF, Downey DG, Van Braeckel E, Rowe SM, Tullis E, et al. VX17-445-103 Study Group. Efficacy and safety of the elexacaftor/tezacaftor/ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomized, phase 3 trial. Lancet. 2019;394:1940–1948. - PMC - PubMed
    1. Middleton PG, Mall MA, Dřevínek P, Lands LC, McKone EF, Polineni D, et al. VX17-445-102 Study Group. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381:1809–1819. - PMC - PubMed

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