2 results
Approved WMOCompleted
Primary Objective(s): To evaluate long-term safety in patients with mucopolysaccharidosis type IIIA disease (MPS IIIA or Sanfilippo Type A) who received HGT-1410Secondary Objective(s):To evaluate:• The long-term cognitive function as measured by the…
Approved WMOCompleted
The purpose of this study is to investigate the safety and efficacy of rFIXFc in previously untreated patients (PUPs) in accordance with the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) guideline on clinical…