3 results
Approved WMOCompleted
To investigate the effect of cholic acid supplementation on the clinical and biochemical parameters of Zellweger spectrum disorder
Approved WMOPending
Primary end point• Progression-free survival (PFS) per ICR central assessmentSecondary end point• Objective response rate (ORR)• Disease control rate (DCR)• Overall survival (OS)• PFS according to Investigator assessment of radiologic images•…
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.