3 results
Primary objective:- To evaluate the safety and tolerability of single ascending doses of ASP3652 in healthy young Caucasian male and female subjectsSecondary objectives:- Different pharmacokinetic outcomes- Different pharmacodynamic outcomes
The primary objective of the study is the following:• To characterize the safety and tolerability of DNL310 in pediatric subjects with MPS II (all cohorts)The secondary objectives of the study are as follows:• To characterize the CNS effects of…
The primary objectives are: To evaluate the CNS activity of DNL310 vs idursulfase as measured by the cerebrospinal fluid (CSF) concentration of heparan sulfate (HS) in participants with the neuronopathic form of mucopolysaccharidosis type II (nMPS…