3-methylglutaconic aciduria type 4

MeSH: C565393ORPHA: 67048

Overview

3-methylglutaconic aciduria that is characterized by mild or intermittent urinary excretion of 3-methylglutaconic acid

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 3-methylglutaconic aciduria type 4, sourced from HPO and Orphanet clinical annotations.

MicrocephalyHearing impairmentCataractIntellectual disabilitySeizureHypotoniaSpasticityGlobal developmental delayDecreased liver functionFailure to thriveCardiomyopathyThrombocytopeniaHypoglycemiaDysgenesis of the cerebellar vermisLactic acidosis3-Methylglutaconic aciduriaIris hypopigmentation

Classification & Codes

MeSH Code

C565393

Orphanet Code

ORPHA:67048
3-methylglutaconic aciduria type 4
MeSHC565393
OrphanetORPHA:67048
Treatments0 drug(s)
Symptoms on record17 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
3-methylglutaconic aciduria type 4 | OrphanDrug