Tetrahydrobiopterin-responsive phenylalanine hydroxylase deficiency.

Kure, S; Hou, D C; Ohura, T; Iwamoto, H; Suzuki, S; Sugiyama, N; Sakamoto, O; Fujii, K et al. · J Pediatr · 1999

case_series · Level IV

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Abstract

Serum phenylalanine concentrations decreased in 4 patients with hyperphenylalaninemia after loading with tetrahydrobiopterin. There were no abnormalities in urinary pteridine excretion or in dihydropteridine reductase activity. However, mutations were detected in the phenylalanine hydroxylase gene, suggesting a novel subtype of phenylalanine hydroxylase deficiency that may respond to treatment with cofactor supplementation.

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