3 results
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…
Primary:• To evaluate the long-term safety of PTC923 in subjects with phenylketonuria (PKU)• To evaluate changes from baseline in dietary phenylalanine (Phe)/protein consumptionSecondary:• To evaluate PTC923 effect on quality of life (QOL) using the…
The primary aim of the NOPHO-DBH AML 2012 study is to improve EFS and OS in children with AML. To improve outcome, an intensified induction regimen will be given and a response guided risk-group stratification using flow cytometric minimal residual…