4 results
To assess the safety, tolerability and the effects of treatment on ocular outcomes following a single intravitreal administration of ESBA1008 compared to Lucentis in patients with exudative AMD
The objective of this study is to evaluate the possible effect of arginine on galactose oxidative capacity in 5 patients with classic galactosemia (homozygous for the p.Q188R mutation).
PrimaryThe primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH).The primary objective for Part B of the study is to demonstrate a…
The primary objective of the study is:* To demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab 15 mg/kg intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial…