Search

Sepiapterin (Sephience; PTC Therapeutics Inc.) for Phenylketonuria

An oral phenylalanine hydroxylase (PAH) activator for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU), to be used in conjunction with a phenylalanine-restricted diet.

Complete this form to request the full report