2 results
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).
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…