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Neumol Pediatr 2018; 13 (3): 118 - 121

Progresos en farmacoterapia en fibrosis quística

fibrosis who have the Gly551Asp-CFTR mutation: a phase

3, open-label extension study (PERSIST). Lancet Respir Med

2014 ;2(11):902–10.

14. Van Goor F, Hadida S, Grootenhuis PDJ, Burton B, Cao

D, Neuberger T et al. Rescue of CF airway epithelial cell

function in vitro by a CFTR potentiator, VX-770. Proc Natl

Acad Sci 2009 Nov 3;106(44):18825–30.

15. Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC,

Dřevínek P et al. A CFTR Potentiator in Patients with

Cystic Fibrosis and the G551D Mutation. N Engl J Med

2011;365(18):1663–72.

16. Davies JC, Wainwright CE, Canny GJ, Chilvers MA,

Howenstine MS, Munck A, et al. Efficacy and safety of

ivacaftor in patients aged 6 to 11 years with cystic fibrosis

with a G551D mutation. Am J Respir Crit Care Med

2013;187(11):1219–25.

17. Davies JC, Cunningham S, Harris WT, Lapey A, Regelmann

WE, Sawicki GS et al. Safety, pharmacokinetics, and

pharmacodynamics of ivacaftor in patients aged 2–5

years with cystic fibrosis and a CFTR gating mutation

(KIWI): an open-label, single-arm study. Lancet Respir Med

2016;4(2):107–15.

18. Patel S, Sinha IP, Dwan K, Echevarria C, Schechter M,

Southern KW. Potentiators (specific therapies for class III

and IV mutations) for cystic fibrosis. Cochrane database Syst

Rev 2015 Mar 26;(3):CD009841.

19. Solomon GM, Marshall SG, Ramsey BW, Rowe SM.

Breakthrough therapies: Cystic fibrosis (CF) potentiators and

correctors. Pediatr Pulmonol 2015;50(S40):S3–13.

20. Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang

X, Cipolli M et al. Lumacaftor–Ivacaftor in Patients with

Cystic Fibrosis Homozygous for Phe508del CFTR. N Engl J

Med 2015;373(3):220–31.

21. Van Goor F, Hadida S, Grootenhuis PDJ, Burton B, Stack JH,

Straley KS et al. Correction of the F508del-CFTR protein

processing defect in vitro by the investigational drug VX-

809. Proc Natl Acad Sci U S A 2011;108(46):18843–8.

22. Milla CE, Ratjen F, Marigowda G, Liu F, Waltz D, Rosenfeld

M. Lumacaftor/Ivacaftor in Patients Aged 6–11 Years with

Cystic Fibrosis and Homozygous for F508del-CFTR. Am J

Respir Crit Care Med 2017;195(7):912–20.

23. Ratjen F, Hug C, Marigowda G, Tian S, Huang X, Stanojevic

S et al. Efficacy and safety of lumacaftor and ivacaftor in

patients aged 6–11 years with cystic fibrosis homozygous

for F508del-CFTR : a randomised, placebo-controlled phase

3 trial. Lancet Respir Med 2017;5(7):557–67.

24. Aslam A, Jahnke N, Remmington T, Southern KW. Ataluren

and similar compounds (specific therapies for premature

termination codon class I mutations) for cystic fibrosis.

Paediatr Respir Rev 2017;24(1):32–4.

25. Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ,

Trifillis P et al. PTC124 targets genetic disorders caused by

nonsense mutations. Nature 2007;447(7140):87–91.

26. Lopes-Pacheco M. CFTR Modulators: Shedding Light on

Precision Medicine for Cystic Fibrosis. Front Pharmacol

2016;7:1–20.

27. Zhang W, Zhang X, Zhang YH, Strokes DC, Naren AP.

Lumacaftor/ivacaftor combination for cystic fibrosis patients

homozygous for Phe508del-CFTR. Drugs Today (Barc) 2016

;52(4):229–37.