5 results
To test the hypothesis that IVIG treatment diminish the risk of development of chronic disease, we designed a prospective clinical intervention study in children with newly diagnosed ITP.
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 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 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…
1. To test the performance of the wearable SDD (Nightwatch) prospectively and the remote SDDs (video and audio) retrospectively in a family home setting. 2. To explore the feasibility of the Nightwatch