2 results
Approved WMOCompleted
* Primary Objective:To assess the effect of MEDI0382 on hepatic glycogen levels versus placebo after 28 days (Part A) and 35 days (Part B) of treatment* Secundary Objective: - To assess the effect of MEDI0382 on hepatic glycogen levels versus…
Approved WMORecruiting
The primary objective of this study is to evaluate the efficacy of olipudase alfa in adult patients with acid sphingomyelinase deficiency.