2 results
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…
Approved WMOCompleted
Objectives: To test the hypothesis that the thyroid gland of offspring of long-lived siblings is more resistant to stimulation with TSH compared to the thyroid gland of their partners, offspring and partners will receive a low dose of recombinant…