6 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…
The aim of this study is to determine whether subcutaneous administration of insulin detemir, as compared to NPH insulin, leads to a more pronounced effect on brain glucose metabolism and blood flow in brain regions associated with appetite…
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 purpose of this study is to explore the efficacy and safety of LTP001 in participants with WHO Group 1 pulmonary hypertension (PH), also referred to as Pulmonary Arterial Hypertension (PAH).This proof of concept study will be executed as the…
To assess the long term safety of LTP001 in participants with pulmonary arterial hypertension (PAH).