3 results
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…
Primary objective:To demonstrate superiority of three dose levels of oral NNC0385-0434 versus placebo on percent change in LDL-C from baseline to week 12 in patients with established ASCVD or ASCVD risk on maximally tolerated statin dose and other…
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…