5 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…
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(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…
In this study, the aim is to perform a home based evaluation of the SENS-UTM Bladder Sensor during the night to examine the usability of the SENS-UTM for ambulatory care in children with nocturnal enuresis.
To compare ACM at Day 42 following treatment with olorofim versus treatment with AmBisome® followed by SOC in the intent-to-treat (ITT) population of patients with IFD caused by proven IA at any site or probable lower respiratory tract disease (LRTD…