PKU is a rare genetic disease that manifests ... Drug treatment is an option for some patients, with BioMarin's enzyme-based therapies Kuvan (sapropterin) or Palynziq (pegvaliase) both approved ...

PKU is a genetic disease that manifests at birth ... namely BioMarin’s enzyme-based therapies Kuvan (sapropterin) or Palynziq (pegvaliase), which are both approved to reduce levels of phe.