phenylketonuria

ICD-10: E70.0MeSH: D010661ORPHA: 716

Overview

amino acid metabolic disorder that is characterized by a mutation in the gene for the hepatic enzyme phenylalanine hydroxylase (PAH), rendering it nonfunctional

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Clinical Presentation

Signs and symptoms associated with phenylketonuria, sourced from HPO and Orphanet clinical annotations.

PhenylalaninuriaMicrocephalyAtypical behaviorOsteopeniaEczematoid dermatitisHypopigmentation of the skinSeizureGlobal developmental delaySpecific learning disabilityGrowth delayEEG abnormalityAbnormal cerebral white matter morphologyHyperphenylalaninemiaSevere intellectual disabilityMusty odorDepressionDementiaShort attention spanAnxietyAtaxiaEncephalopathyTremorLower limb spasticityAbnormal cardiovascular system morphologyCerebral visual impairment

Classification & Codes

ICD-10 Code

E70.0

MeSH Code

D010661

Orphanet Code

ORPHA:716
phenylketonuria
ICD-10E70.0
MeSHD010661
OrphanetORPHA:716
Treatments0 drug(s)
Symptoms on record25 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO