3 results
Approved WMOCompleted
To investigate the effect of cholic acid supplementation on the clinical and biochemical parameters of Zellweger spectrum disorder
Approved WMORecruiting
Investigating the long-term safety of personalized cholic acid treatment in patients with defects in bile acid synthesis based on clinical and biochemical parameters.
Approved WMOCompleted
Primary:The primary objective of this study is to evaluate the efficacy of LDX administered as a daily morning dose (30, 50, and 70mg/day) compared to placebo over the course of 7 weeks. This study will enrol children and adolescents (6-17 years of…