2 results
Approved WMORecruiting
The primary objective of this study is to evaluate the efficacy of olipudase alfa in adult patients with acid sphingomyelinase deficiency.
Approved WMORecruiting
The co-primary endpoints of this study are PFS in intermediate and poor-risk subjects, as assessed by an Independent Radiology Review Committee (IRRC) and OS in intermediate and poor-risk subjects. The final analysis of PFS will occur after 583…