2 results
Approved WMOCompleted
Main objective: The main objective of this open-label, single arm study is to further characterize the safety, tolerability and effectiveness profile of olesoxime in SMAThe primary safety objective for this study is as follows:To evaluate the safety…
Approved WMOCompleted
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…