BioMarin BMRN has announced positive results from the phase III PEGASUS study evaluating Palynziq (pegvaliase) in adolescents aged 12-17 years with phenylketonuria (PKU). The study achieved its ...

Shares in Synlogic have halved in value after the company reported that a phase 3 trial of its main pipeline drug for rare metabolic disorder phenylketonuria (PKU) failed to show efficacy ...

PKU is a genetic disease that manifests at birth and results from an inability to break down phenylalanine (phe), an amino acid that is commonly found in many foods. Left untreated, high levels of ...

PKU is a rare genetic disorder that prevents ... leading to toxic levels that can cause severe neurological damage. While newborn screening enables early diagnosis, lifelong dietary management ...