3 results
Primary objective:To characterize the clearance pathways, the route of excretion and the total recovery of radioactivity, PBI-4050 and its major metabolites in healthy subjects following administration of a single oral dose of 1200 mg [14C]-PBI-4050…
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…