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 WMOCompleted
The primary objective is to evaluate the inhalation process and the overall satisfaction with the use of the Investigational Medicinal Products (IMPs). The secondary objectives are to determine the subjective effects after pulmonary administration…