3 results
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…
Primary ObjectiveThe primary objective of this study is to evaluate the effect of 12 weeks of treatment with 2 different doses of oral AQX-1125 (100 mg or 200 mg) administered once daily compared to placebo on the change from Baseline (Visit 2) to…
To evaluate the effectiveness and safety of concentration controlled combination of once daily dosed low-dose sirolimus (trough levels: 3-5 ng/ml) and extended-release tacrolimus (trough levels:3-5 ng/ml), in order to provide superior renal function…