3 results
The aim of this study is to explore the value and added value of a wrist unit that can be put in a flexion position (Flex-wrist, Otto Bock ®) and a wrist unit that can not only be put in a flexion position but can also move freely in a neutral…
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…
Primary: To characterize the safety and tolerability of PDR001 in combination with LCL161, everolimus, HDM201, QBM076 or panobinostat and to identify recommended doses and schedules for future studies.Secondary: To characterize changes in the immune…