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 demonstrate non-inferiority of a T2T strategy in which conventional synthetic disease modifying drugs (csDMARDs) refractory RApatients are initially treated with tsDMARD baricitinib versus the comparable T2T strategy in which patients are…