Malonyl-CoA decarboxylase deficiency

MeSH: C535702ORPHA: 943

Overview

Malonic aciduria is a metabolic disorder caused by deficiency of malonyl-CoA decarboxylase (MCD)

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 Malonyl-CoA decarboxylase deficiency, sourced from HPO and Orphanet clinical annotations.

Delayed speech and language developmentDystoniaMethylmalonic aciduriaElevated urine malonic acid levelMicrocephalySeizureGlobal developmental delayFailure to thriveGrowth delayCardiomyopathyMetabolic acidosisHypoglycemiaFeeding difficultiesReduced malonyl-CoA decarboxylase activity in cultured fibroblastsHypotoniaLethargyKetosisVomitingDiarrhea

Classification & Codes

MeSH Code

C535702

Orphanet Code

ORPHA:943
Malonyl-CoA decarboxylase deficiency
MeSHC535702
OrphanetORPHA:943
Treatments0 drug(s)
Symptoms on record19 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO