Fig. 4
Diagrammatic representation of the course of a successful therapy trial with sapropterin in a newborn with BH4-responsive PAH deficiency. BH4: Tetrahydrobiopterin; PAH: Phenylalanine hydroxylase; Phe: Phenylalanine; PKU: Phenylketonuria
Fig. 4 | Orphanet Journal of Rare Diseases