2 results
Approved WMORecruiting
The primary aim is to examine whether early addition of IVIg to standard treatment with prednisone in patients with newly diagnosed myositis leads to superior clinical outcome after 12 weeks. Our secondary aims are to examine the effect the…
Approved WMORecruiting
Primary Objective (Cohort 1)To demonstrate a sustained effect of A4250 on serum bile acids and pruritus in children with progressive familial intrahepatic cholestasis (PFIC) Types 1 and 2.Primary Objective (Cohort 2)To evaluate the effect of A4250…