2 results
Approved WMOCompleted
Primary:• To evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria (PKU) as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (ie, the average of each respective…
Approved WMOCompleted
The main objective is to determine the efficacy of Lyumjev in the Inreda AP system. Secondary objectives are: to assess safety, differences in pharmacodynamics and differences in AP-related outcome measures.