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 WMOWill not start
The primary objective of this study is to determine if ambrisentan is effective in delaying disease progression and death in subjects with IPF.Secondary objectives include evaluation of the safety and effect of ambrisentan on development of…