论文信息 - Trying to find a cure for cystic fibrosis: CFTR biomarkers as outcomes

Trying to find a cure for cystic fibrosis: CFTR biomarkers as outcomes

To the Editor: In a recent issue of the European Respiratory Journal , De Boeck et al. 1 provided a systematic review of the clinimetric properties of cystic fibrosis transmembrane conductance regulator (CFTR) biomarkers, giving supporting evidence for promoting these biomarkers as surrogate end-points. The authors are to be congratulated not only for having collected exhaustive data about CFTR biomarkers and attempted to clarify their role but also for having led tremendous efforts towards standardisation of …

P. Lebecque

[1] M. Amaral,et al. CFTR biomarkers: time for promotion to surrogate end-point , 2012, European Respiratory Journal.

[2] J. Clancy,et al. 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 , 2011, Thorax.

[3] M S Pepe,et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. , 1999, The New England journal of medicine.