2 results
Approved WMORecruiting
Primary objectives:- To confirm the haemostatic effect of Mim8 as treatment prophylaxis for adult and adolescent patients with haemophilia A with or without inhibitors.Secondary objectives:- To investigate safety of Mim8 prophylaxis in adults and…
Approved WMORecruiting
The primary objective of this study is to evaluate the efficacy of olipudase alfa in adult patients with acid sphingomyelinase deficiency.