3 results
Main objectivePart A: To evaluate the safety and efficacy of treatment with VX-371, administered with and without 4.2% hypertonic saline (HS) in subjects with primary ciliary dyskinesia (PCD) who are *12 years of age.Part B: To evaluate the safety…
Primary Objective* To compare the effect of a 30-week course of mavacamten with placebo on clinical response comprising of exercise capacity and clinical symptoms in participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM)…
Main objective- To characterize the effect of GLPG2737 on growth in total kidneyvolume (TKV) compared to placebo.- To evaluate the safety and tolerability of oral doses of GLPG2737compared to placebo.Secondary objectives: - To characterize the…