4 results
To investigate the effect of cholic acid supplementation on the clinical and biochemical parameters of Zellweger spectrum disorder
The objective of this study is to assess the performance of the Masimo O3 regional oximeter on somatic tissue oxygenation, under controlled hypoxia. Somatic rSO2 values will be compared with combined arterial and venous blood gas measurements from…
The primary objectives of the study are to:Determine the OBD(s) and RP2D's) of BMF-219 monotherapy administered daily based on evaluation of all available PK/ PD, target engagement, safety, and tolerability data. Note that the OBD and RP2D may…
Investigating the long-term safety of personalized cholic acid treatment in patients with defects in bile acid synthesis based on clinical and biochemical parameters.