1. Stoltz DA, Meyerholz DK, Welsh MJ. Origins of cystic fibrosis lung disease. N Engl J Med 2015;372:1574–1575. 2. Quon BS, Rowe SM. New and emerging targeted therapies for cystic… Click to show full abstract
1. Stoltz DA, Meyerholz DK, Welsh MJ. Origins of cystic fibrosis lung disease. N Engl J Med 2015;372:1574–1575. 2. Quon BS, Rowe SM. New and emerging targeted therapies for cystic fibrosis. BMJ 2016;352:i859. 3. Accurso FJ, Van Goor F, Zha J, Stone AJ, Dong Q, Ordonez CL, et al. Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data. J Cyst Fibros 2014;13:139–147. 4. McKone EF, Velentgas P, Swenson AJ, Goss CH. Association of sweat chloride concentration at time of diagnosis and CFTR genotype with mortality and cystic fibrosis phenotype. J Cyst Fibros 2015;14:580–586. 5. Rowe SM, Verkman AS. Cystic fibrosis transmembrane regulator correctors and potentiators. Cold Spring Harb Perspect Med 2013;3:pii: a009761. 6. McCague AF, Raraigh KS, Pellicore MJ, Davis-Marcisak EF, Evans TA, Han ST, et al. Correlating cystic fibrosis transmembrane conductance regulator function with clinical features to inform precision treatment of cystic fibrosis. Am J Respir Crit Care Med 2019;199:1116–1126. 7. Clinical and Functional Translation of CFTR (CFTR2). Welcome to the CFTR2 website [accessed 2019 Mar 25]. Available from: http://cftr2.org. 8. Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Dřevı́nek P, et al.; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011;365:1663–1672. 9. Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, et al.; TRAFFIC Study Group; TRANSPORT Study Group. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med 2015;373:220–231. 10. Taylor-Cousar JL, Munck A, McKone EF, van der Ent CK, Moeller A, Simard C, et al. Tezacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del. N Engl J Med 2017;377:2013–2023. 11. Davies JC, Moskowitz SM, Brown C, Horsley A, Mall MA, McKone EF, et al.; VX16-659-101 Study Group. VX-659-tezacaftor-ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles. N Engl J Med 2018;379:1599–1611. 12. Keating D, Marigowda G, Burr L, Daines C, Mall MA, McKone EF, et al.; VX16-445-001 Study Group. VX-445-tezacaftor-ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles. N Engl J Med 2018;379:1612–1620. 13. Vanscoy LL, Blackman SM, Collaco JM, Bowers A, Lai T, Naughton K, et al. Heritability of lung disease severity in cystic fibrosis. Am J Respir Crit Care Med 2007;175:1036–1043. 14. Collaco JM, Blackman SM, McGready J, Naughton KM, Cutting GR. Quantification of the relative contribution of environmental and genetic factors to variation in cystic fibrosis lung function. J Pediatr 2010;157:802–7.e1, 3. 15. Saiman L, Mayer-Hamblett N, Campbell P, Marshall BC; Macrolide Study Group. Heterogeneity of treatment response to azithromycin in patients with cystic fibrosis. Am J Respir Crit Care Med 2005;172: 1008–1012.
               
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