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 WMORecruiting
To assess the feasibility of using a subtherapeutic dose of piracetam as a marker of adherence to therapy by determining whether this subtherapeutic dose produces a detectable concentration in the urine