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Year to year change in FEV(1) in patients with cystic fibrosis and different mutation classes.
De Boeck K, Zolin A. De Boeck K, et al. J Cyst Fibros. 2017 Mar;16(2):239-245. doi: 10.1016/j.jcf.2016.09.009. Epub 2016 Oct 11. J Cyst Fibros. 2017. PMID: 27745801 Free article.
In patients with cystic fibrosis, most treatments addressing the underlying basic defect are mutation or mutation class specific. ...Whereas patients with at least one mutation of class IV or V have on average a lower …
In patients with cystic fibrosis, most treatments addressing the underlying basic defect are mutation or muta
Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis.
Whiting P, Al M, Burgers L, Westwood M, Ryder S, Hoogendoorn M, Armstrong N, Allen A, Severens H, Kleijnen J. Whiting P, et al. Health Technol Assess. 2014 Mar;18(18):1-106. doi: 10.3310/hta18180. Health Technol Assess. 2014. PMID: 24656117 Free PMC article.
BACKGROUND: Ivacaftor (Kalydeco( ), Vertex Pharmaceuticals) is the first of a new class of drugs that target the underlying protein defect in cystic fibrosis (CF). It is aimed at patients with the G551D (glycine to aspartate change in nucleotide …
BACKGROUND: Ivacaftor (Kalydeco( ), Vertex Pharmaceuticals) is the first of a new class of drugs that target the underlying protein d …
A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis.
Alton EWFW, Armstrong DK, Ashby D, Bayfield KJ, Bilton D, Bloomfield EV, Boyd AC, Brand J, Buchan R, Calcedo R, Carvelli P, Chan M, Cheng SH, Collie DS, Cunningham S, Davidson HE, Davies G, Davies JC, Davies LA, Dewar MH, Doherty A, Donovan J, Dwyer NS, Elgmati HI, Featherstone RF, Gavino J, Gea-Sorli S, Geddes DM, Gibson JSR, Gill DR, Greening AP, Griesenbach U, Hansell DM, Harman K, Higgins TE, Hodges SL, Hyde SC, Hyndman L, Innes JA, Jacob J, Jones N, Keogh BF, Limberis MP, Lloyd-Evans P, Maclean AW, Manvell MC, McCormick D, McGovern M, McLachlan G, Meng C, Montero MA, Milligan H, Moyce LJ, Murray GD, Nicholson AG, Osadolor T, Parra-Leiton J, Porteous DJ, Pringle IA, Punch EK, Pytel KM, Quittner AL, Rivellini G, Saunders CJ, Scheule RK, Sheard S, Simmonds NJ, Smith K, Smith SN, Soussi N, Soussi S, Spearing EJ, Stevenson BJ, Sumner-Jones SG, Turkkila M, Ureta RP, Waller MD, Wasowicz MY, Wilson JM, Wolstenholme-Hogg P; on behalf of the UK Cystic Fibrosis Gene Therapy Consortium. Alton EWFW, et al. Southampton (UK): NIHR Journals Library; 2016 Jul. Southampton (UK): NIHR Journals Library; 2016 Jul. PMID: 27441329 Free Books & Documents. Review.
BACKGROUND: Cystic fibrosis (CF) is a chronic, life-limiting disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene leading to abnormal airway surface ion transport, chronic lung infections, inflammation and eventual respiratory …
BACKGROUND: Cystic fibrosis (CF) is a chronic, life-limiting disease caused by mutations in the CF transmembrane conduc …