3 results
Approved WMOCompleted
To investigate the effect of cholic acid supplementation on the clinical and biochemical parameters of Zellweger spectrum disorder
Approved WMORecruiting
Dose escalation phase:Primary• Determine the RP2D and if reached, the MTD of GEN3014• Evaluate the safety and tolerability of GEN3014 Secondary:• Characterize the PK properties of GEN3014• Characterize the pharmacodynamic properties of GEN3014•…
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.