3 results
Approved WMOCompleted
To investigate the effect of cholic acid supplementation on the clinical and biochemical parameters of Zellweger spectrum disorder
Approved WMOCompleted
The study aims to limit the use of anthracyclines and to reduce the dose of ATRA. Another aim is to stratify treatment by risk group: standard risk - WBC <10 x 109/l : high risk - WBC >=10 x 109/l. Furthermore this study aims to…
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.