2 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
The objective of this study is to determine the efficacy, safety, and tolerability of tafamidis in subjects with transthyretin cardiomyopathy.The primary objective is to assess the efficacy, safety, and tolerability of an oral dose of 20 mg or 80 mg…