3 results
Approved WMOCompleted
The primary objective of this study is to examine the direct influence of the grasp support system on (functional) performance and movement execution of the most-affected arm and hand. Secondary objectives are to examine the direct effect of the…
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.
Approved WMORecruiting
The objectives of this are to establish the safety, tolerability, and efficacy of VRDN-001, and the pharmacokinetic (PK) and pharmacodynamic (PD) profiles of VRDN-001 in HV and TED patients over a dose range of 3.0 to 20.0 mg/kg.