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A randomised, double-blind study evaluating the safety, tolerability, protein accretion, amino acid plasma levels and long-term outcome of Neoven compared to Vaminolact in premature very low birth weigth (VLBW) infants.

To evaluate the safety, tolerability, protein accretion, amino acid plasma levels and long-term outcome of Neoven compared to Vaminolact in preterm infants with a birth weight from 800 g to less than 1500 g and with a gestational age from 25 weeks…

A phase 3 study of PTC923 in subjects with phenylketonuria

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…

A phase 3 open-label study of PTC923 (Sepiapterin) in Phenylketonuria

This study has been transitioned to CTIS with ID 2023-509229-31-00 check the CTIS register for the current data. Primary:• To evaluate the long-term safety of PTC923 in subjects with phenylketonuria (PKU)• To evaluate changes from baseline in…