branched-chain keto acid dehydrogenase kinase deficiency

ORPHA: 308410

Overview

autosomal recessive disease characterized by autism, epilepsy, intellectual disability, and reduced levels of branched-chain amino acids that has material basis in homozygous mutation in the BCKDK gene on chromosome 16p11

Available Treatments (0)

No treatments linked yet

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

Classification & Codes

Orphanet Code

ORPHA:308410
branched-chain keto acid dehydrogenase kinase deficiency
OrphanetORPHA:308410
Treatments0 drug(s)
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
branched-chain keto acid dehydrogenase kinase deficiency | OrphanDrug