Tetrahydrobiopterin-responsive hyperphenylalaninemia/phenylketonuria

Orpha code: 293284OMIM code:

Definition

A form of phenylketonuria (PKU), an inborn error of amino acid metabolism, characterized by mild to moderate symptoms of PKU including impaired cognitive function, seizures, and behavioral and developmental disorders, and a marked reduction of elevated phenylalanine concentrations after oral loading with tetrahydrobiopterin (BH4), an essential cofactor of phenylalanine hydroxylase.

Disease data
Classification

Clinical subtype

Synonyms
BH4-responsive HPA/PKU
Hiperfenyloalaninemia/fenyloketonuria wrażliwa na BH4
HPA/PKU wrażliwa na BH4
HPA/PKU wrażliwa na tetrahydrobiopterynę
BH4-responsive hyperphenylalaninemia/phenylketonuria
Tetrahydrobiopterin-responsive HPA/PKU
ORPHA code
293284
OMIM code
-
ICD10 code
E70.1
ICD11 code
-

No additional description.

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