4 results
The purpose of the study is to investigate how quickly and to what extent radiprodil is absorbed and eliminated from the body (this is called pharmacokinetics). It will also be investigated to what extent radiprodil is tolerated. In addition, 2 new…
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…
This project aims to evaluate the impact of an intensive dietetic care pathway on dietary protein intake and physical functioning in older adults during hospitalisation and post-discharge. In addition, the cost-effectiveness of this intensive…
Primary Objective:-To determine the long-term safety and tolerability of multiple individually titrated doses of radiprodil as an addon therapy to standard of care (SOC)in pediatric participants-To establish a safe and well tolerated dose after 8…