Ivacaftor-treated Patients with Cystic Fibrosis Derive Long-Term Benefit Despite No Short-Term Clinical Improvement
- PMID: 29256624
- PMCID: PMC6005553
- DOI: 10.1164/rccm.201710-2046LE
Ivacaftor-treated Patients with Cystic Fibrosis Derive Long-Term Benefit Despite No Short-Term Clinical Improvement
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References
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- Rowe SM, Heltshe SL, Gonska T, Donaldson SH, Borowitz D, Gelfond D, et al. GOAL Investigators of the Cystic Fibrosis Foundation Therapeutics Development Network. Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis. Am J Respir Crit Care Med. 2014;190:175–184. - PMC - PubMed
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- De Boeck K, Munck A, Walker S, Faro A, Hiatt P, Gilmartin G, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros. 2014;13:674–680. - PubMed
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- Sawicki GS, McKone EF, Pasta DJ, Millar SJ, Wagener JS, Johnson CA, et al. Sustained benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data. Am J Respir Crit Care Med. 2015;192:836–842. - PubMed
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- Knapp EA, Fink AK, Goss CH, Sewall A, Ostrenga J, Dowd C, et al. The Cystic Fibrosis Foundation Patient Registry. design and methods of a national observational disease registry. Ann Am Thorac Soc. 2016;13:1173–1179. - PubMed
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