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 aim of the trial is to demonstrate the non-inferiority of AMT-061 (2 × 1013 gc/kg) during the 52 weeks following establishment of stable factor IX expression (months 6 to 18) post-treatment (AMT-061) follow-up compared to standard of…