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 WMOPending
Part 1:To evaluate the effects of 52 weeks of treatment with basmisanil on core symptom domains of Dup15q syndrome (language and social skills) and dailyfunctioning. Part 2:• To evaluate the tolerability and safety of up to 3 years of treatment with…