BH4-deficient hyperphenylalaninemia C

ORPHA: 226

Overview

genetic metabolic disorder

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 BH4-deficient hyperphenylalaninemia C, sourced from HPO and Orphanet clinical annotations.

MicrocephalyIntellectual disabilityGlobal developmental delayDysphagia

Classification & Codes

Orphanet Code

ORPHA:226
BH4-deficient hyperphenylalaninemia C
OrphanetORPHA:226
Treatments0 drug(s)
Symptoms on record4 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO