Ballicules.com

PAH activator

• Oral precursor of tetrahydrobiopterin (BH4), the cofactor phenylalanine hydroxylase requires
• Restores residual PAH activity in responsive phenylketonuria, lowering blood phenylalanine
• Response depends on residual enzyme — null PAH variants do not respond

Drugs with this mechanism (1)

• Sepiapterin — BH4 precursor for phenylketonuria; restores cofactor needed by phenylalanine hydroxylase.

All mechanisms · Open the interactive console ↗