3 results
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.
Primary Objective:The primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP).The primary efficacy objective of the study is to assess…
Primary ObjectiveThe primary efficacy objective of the study is to assess the effect of garetosmab (10 mg/kg) versus placebo on the formation of new HO lesions from baseline to week 56, as determined by low dose CT.The primary safety objective of…