3 results
Primary objective- To measure the effect of sapropterin on diurnal and day to day variations of blood phenylalanine concentrations.Secondary objective- To measure the effect of sapropterin on diurnal and day to day variations of blood tyrosine…
In this study we will investigate how quickly and to what extent ganaplacide is absorbed, transported, and eliminated from the body. Ganaplacide will be radioactively labelled with carbon-14 (14C). In this way, ganaplacide can be traced in blood,…
To assess the ability of MRI techniques to detect early stages of lipid accumulation in the liver of ASMD patients with the chronic visceral subtype compared to values of age-, sex- and BMI-matched healthy subjects.