5 results
The primary objectives of this study is:* To determine the safety and tolerability of rhHNS via ascending doses administered via a surgically implanted intrathecal drug device (IDDD) once monthly for 6 months, in patientswith MPS IIIA.The secondary…
The primary goal of the study is to assess the safety and performance of the Treovance Stent-Graft with Navitel Delivery System in subjects with infrarenal aortic aneurysms, specifically to evaluate if the diseased pathology can be treated with an…
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…
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…
The primary objective is to compare the effectiveness of ambroxol versus placebo on the MDS-UPDRS part III motor sub-score in the *practically defined OFF-medication state* in patients with moderate PD, carrying a GBA mutation. The hypothesis is…