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 WMORecruiting
Primary objective: To investigate the safety of Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.Secondary objectives• To investigate the efficacy of Mim8 prophylaxis in children with haemophilia A with or without…