4 results
Primary objectives of this study:1. Evaluate the efficacy after 26 weeks of Kuvan® treatment + Phe-restricted diet therapy in increasing dietary Phe tolerance, as compared to dietary therapy alone in <4 year-old infants and children with…
To investigate the effect of cholic acid supplementation on the clinical and biochemical parameters of Zellweger spectrum disorder
Primary objective: To evaluate the expected increase of the Microvascular Flow Index perioperative using a SDF-imaging device and the systemic inflammatory response by measuring inflammation biomarkers.Secondary objective: To learn more about the…
Investigating the long-term safety of personalized cholic acid treatment in patients with defects in bile acid synthesis based on clinical and biochemical parameters.