2 results
Approved WMOCompleted
Primary objective:To evaluate the efficacy of JZP-258 in the treatment of cataplexy in subjects with narcolepsyKey Secondary objective:To evaluate the efficacy of JZP-258 in the treatment of excessive daytime sleepiness (EDS) in…
Approved WMOWill not start
The purpose of this study is to evaluate the possible risks and efficacy (improvement of disease) with an experimental oral study drug named eliglustat in pediatric patients from 2 to 18 years with Gaucher disease.