3 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
The objectives of the study are:* To evaluate the safety and tolerability of zilucoplan in subjects with IMNM* To evaluate the efficacy of zilucoplan in subjects with IMNM
Approved WMOCompleted
To evaluatie the long term safety of treatment with osilodrostat for patients with Cushing Syndrome. In addition to evaluating the proportion of patients with clinical benefit (as assessed by the investigator) and determening frequency, severity and…