2 results
Approved WMORecruiting
The primary objective is to identify which coagulation factors are associated with unsuccessful reversal of VKA activity. Secondary objectives include the assessment of the number of patients reaching normalized INR (i.e. <1.8) and the number…
Approved WMOCompleted
The primary objectives of this study are: • To determine the longterm safety and tolerability of rhHNS administered via an intrathecal (IT) route once monthly for 8 years in patients with MPS IIIA, who have received and tolerated 6 months of…