2 results
Approved WMOCompleted
Primary• To assess the effects of RVT-101 versus placebo on global function as measured by the Clinician*s Interview-Based Impression of Change Plus Caregiver Input (CIBIC+) after 24 weeks of treatment• To assess the effects of RVT-101 versus…
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…