2 results
Approved WMOCompleted
To demonstrate the efficacy of repeated daily doses of 40 µg/kg/day and 120 µg/kg/day A4250 in children with progressive familial intrahepatic cholestasis (PFIC) Types 1 and 2, as determined by the following:* Proportion of patients experiencing at…
Approved WMORecruiting
To study the effect of voxelotor on the hemodynamics of the cerebral vasculature (CBF and CVR)