2 results
Approved WMOPending
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab compared with placebo in each of the following cohorts: • NMDAR autoimmune encephalitis (AIE) cohort: adults and adolescents (12-16) with definite…
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.