3 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
To improve overall survival of patients with a early-stage high grade uterine leiomyosarcoma.
Approved WMOCompleted
* To investigate the long-term safety and tolerability of ponesimod * To investigate the long-term efficacy of ponesimod* To explore the dose response relationship of 10, 20 and 40 mg ponesimod on lymphocyte count, MRI endpoints and annualized…