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 primary objective of RIN-PF-303 is to evaluate superiority of inhaled treprostinil against placebo for the annual rate of change in absolute forced vital capacity (FVC) from baseline to Week 52.