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
To evaluate the efficacy (superiority) and safety of BAY 41-6551 as measured by the comparison of the clinical cure rate of aerosolized BAY 41-6551, administered via the PDDS Clinical, versus placebo (normal saline) at the Test-of-Cure (TOC0 visit…