2 results
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 WMOPending
Cognitive and functional abilities will be evaluated using psychometric scales (ie, cognitive subscale of the Alzheimer*s Disease Assessment Scale [ADAS cog11]), assessment of activities of daily living (ie, Amsterdam Instrumental Activities of…