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
To test whether monthly infusions (every four weeks) of intravenous Flebogamma® 5% DIF in a 1 year treatment period in PPS subjects are superior to placebo by assessing physical performance, as measured by 2MWD.For Stage 1, to select the optimal…