2 results
A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase deficiency
The primary objective of this study is to evaluate the safety of sebelipase alfa in a more broad population of subjects with LAL Deficiency than have been previously studied.The secondary objectives of this study are (1) to evaluate effects of…
Approved WMOCompleted
PRIMARY* To demonstrate the non-inferiority of intravenous-to-oral antibiotic switch therapy in clinically stable neonates with probable bacterial infection compared to a complete course of intravenous antibiotic therapySECONDARY* To describe the…