2 results
Approved WMORecruiting
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…
Approved WMOCompleted
Primary* To evaluate safety and tolerability of LNP3794 at selected dose levelsSecondary* To determine the therapeutic relevant dose(s) of LNP3794* To describe the PK profile of LNP3794 following single and multiple oral administrationOther* To…