2 results
Not approvedWill not start
The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB074 in subjects with neuropathic PLSR.
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.