Comparison of Organoid Swelling and In Vivo Biomarkers of CFTR Function to Determine Effects of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for the F508del Mutation.
Simon Y GraeberPeter van MourikAnnelotte M VonkEvelien KruisselbrinkStephanie HirtzCornelis K van der EntMarcus Alexander MallJeffrey M BeekmanPublished in: American journal of respiratory and critical care medicine (2020)