2 results
Approved WMOCompleted
Primary objectives: 1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and2. To evaluate the safety and…
Approved WMORecruiting
Primary objective: To investigate the safety of Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.Secondary objectives• To investigate the efficacy of Mim8 prophylaxis in children with haemophilia A with or without…