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 WMORecruiting
The objective of the proposed study is to confirm the findings of the dose ranging trial by, primarily, assessing the effect of two doses of CHF6001 on the rate reduction of moderate and severe exacerbations, when added onto maintenance tripleā¦