

C o n t e n i d o d i s p o n i b l e e n h t t p : / / www. n e umo l o g i a - p e d i a t r i c a . cl
121
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.