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…
To evaluate safety, tolerability pharmacokinetic profile; to explore pharmacodynamics; to evaluate major effects of Guanabenz on quantitative MRI and clinical parameters in pediatric patients with VWM; explore biomarkers in blood and cerebrospinal…
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…