3 results
Approved WMOCompleted
The primary objective of this study is to assess the potential clinical efficacy of HGT-1410administered via a surgically implanted IDDD in patients with MPS IIIA. Efficacy will bemeasured as a meaningful amelioration in the progression of cognitive…
Approved WMOCompleted
In this study we will therefore investigate the influence of hypoxia and anti oxidants on HVR and HPV.
Approved WMOCompleted
The objective of this study is to evaluate the possible effect of arginine on galactose oxidative capacity in 5 patients with classic galactosemia (homozygous for the p.Q188R mutation).