2 results
Approved WMOCompleted
The primary objective of this study is to determine the effect of once-daily oral MGL-3196 60 mgor 100 mg versus placebo for 12 weeks on the percent change from baseline in low-densitylipoprotein cholesterol (LDL-C) in patients with HeFH.Theā¦
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.