HSD10 disease, infantile type

ORPHA: 391428

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 HSD10 disease, infantile type, sourced from HPO and Orphanet clinical annotations.

Hyperkinetic movementsLoss of ambulationDiffuse cerebral atrophyGastrointestinal dysmotilityFrontotemporal cerebral atrophyNonprogressive encephalopathySevere intellectual disabilityAbnormality of the lower urinary tractElevated brain lactate level by MRSSpoken word recognition deficitIncreased circulating lactate concentrationIncreased CSF lactateAbnormality of mitochondrial metabolismAbnormal circulating enzyme concentration or activityBlindnessDelayed speech and language developmentSeizureHypotoniaGlobal developmental delayMetabolic acidosisHypoglycemiaCerebral atrophyNeurodegenerationDevelopmental regressionLactic acidosisModerate global developmental delayAbnormal concentration of acylcarnitine in the urineMicrocephalyHearing impairmentRod-cone dystrophyRetinal degenerationVisual lossNystagmusOptic atrophyRestlessnessParoxysmal bursts of laughterCyanosisDysarthriaSpastic diplegiaChoreoathetosisSpastic tetraparesisDystoniaAbsent speechHypertrophic cardiomyopathyCardiomegalyHyperammonemiaAbnormal facial shapeDysphagiaAbnormal basal ganglia morphologyIncoordinationPoor head control

Classification & Codes

Orphanet Code

ORPHA:391428
HSD10 disease, infantile type
OrphanetORPHA:391428
Treatments0 drug(s)
Symptoms on record51 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO