4 results
To evaluate the safety and tolerability of FFP104 in PBC subjects following repeat doses of FFP104
To evaluate the safety and efficacy of intravenously administered FFP104 in subjects with active Crohn*s disease following repeat doses of FFP104.
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…
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…