2 results
Approved WMOCompleted
The objectives of the study are to:* Identify a dose and dosing regimen of KRN23, based on safety and PD effect, in pediatric XLH patients* Establish the safety profile of KRN23 for the treatment of children with XLH including ectopic mineralization…
Approved WMOCompleted
Primary Core: to demonstrate the efficacy of BAF312 relative to placebo in delaying the time to 3-month confirmed disability progression, measured by EDSS. Main secondary objectives Core: to demonstrate the efficacy in delaying the time to 3-month…