4 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…
The primary objective of this study is to examine the effectiveness of the use of a load-reducing orthosis for two months on functioning (using a validated osteoarthritis function questionnaire (WOMAC)) in ten patients with osteoarthritis of the…
The primary objective of this study is to investigate if an orthosis combined with exercise therapy results in less pain and less conversion to surgery than an orthosis alone in patients with CMC-1 OA at three months and one year after treatment…
To assess the influence of dosing guided by AutoK on achieving PK targets and clinical endpoints in intensive care patients with sepsis.