5 results
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 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…
Primary Objective:To investigate whether arterial microcalcification (18F-NaF-PET detected) and macrocalcification (CT detected) are increased in patients with T2D who have macroalbuminuria as compared to patients with normoalbuminuria.Secondary…
Within the proposed study design it is possible to answer more study questions within the same population and within the same timeframe without adding much burden to the study participants. measurements and analyses will be performed both during a…