Safety and efficacy of the cystic fibrosis transmembrane conductance regulator potentiator icenticaftor (QBW251).
Kazani S, Rowlands DJ, Bottoli I, Milojevic J, Alcantara J, Jones I, Kulmatycki K, Machineni S, Mostovy L, Nicholls I, Nick JA, Rowe SM, Simmonds NJ, Vegesna R, Verheijen J, Danahay H, Gosling M, Ayalavajjala PS, Salman M, Strieter R.
Kazani S, et al. Among authors: rowlands dj.
J Cyst Fibros. 2021 Mar;20(2):250-256. doi: 10.1016/j.jcf.2020.11.002. Epub 2020 Dec 6.
J Cyst Fibros. 2021.
PMID: 33293212
Free PMC article.
Efficacy was assessed in adult CF patients with 1 pre-specified CFTR Class III or IV mutation (150 and 450 mg bid), or homozygous for F508del mutation (450 mg bid). Primary efficacy endpoint was change from baseline in lung clearance index (LCI(2.5)). Secondary endpoints i …
Efficacy was assessed in adult CF patients with 1 pre-specified CFTR Class III or IV mutation (150 and 450 mg bid), or homozygous for F508de …