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Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation.
Clancy JP, Rowe SM, Accurso FJ, Aitken ML, Amin RS, Ashlock MA, Ballmann M, Boyle MP, Bronsveld I, Campbell PW, De Boeck K, Donaldson SH, Dorkin HL, Dunitz JM, Durie PR, Jain M, Leonard A, McCoy KS, Moss RB, Pilewski JM, Rosenbluth DB, Rubenstein RC, Schechter MS, Botfield M, Ordoñez CL, Spencer-Green GT, Vernillet L, Wisseh S, Yen K, Konstan MW. Clancy JP, et al. Among authors: bronsveld i. Thorax. 2012 Jan;67(1):12-8. doi: 10.1136/thoraxjnl-2011-200393. Epub 2011 Aug 8. Thorax. 2012. PMID: 21825083 Free PMC article. Clinical Trial.
Cystic fibrosis transmembrane conductance regulator (CFTR)-mediated residual chloride secretion does not protect against early chronic Pseudomonas aeruginosa infection in F508del homozygous cystic fibrosis patients.
Derichs N, Mekus F, Bronsveld I, Bijman J, Veeze HJ, von der Hardt H, Tummler B, Ballmann M. Derichs N, et al. Among authors: bronsveld i. Pediatr Res. 2004 Jan;55(1):69-75. doi: 10.1203/01.PDR.0000100758.66805.CE. Epub 2003 Nov 6. Pediatr Res. 2004. PMID: 14605249
Diversity of the basic defect of homozygous CFTR mutation genotypes in humans.
Stanke F, Ballmann M, Bronsveld I, Dörk T, Gallati S, Laabs U, Derichs N, Ritzka M, Posselt HG, Harms HK, Griese M, Blau H, Mastella G, Bijman J, Veeze H, Tümmler B. Stanke F, et al. Among authors: bronsveld i. J Med Genet. 2008 Jan;45(1):47-54. doi: 10.1136/jmg.2007.053561. J Med Genet. 2008. PMID: 18178635 Free article.
44 results