Lathosterolosis

MeSH: C537880ORPHA: 46059

Overview

Lathosterolosis is an extremely rare inborn error of sterol biosynthesis characterized by facial dysmorphism, congenital anomalies (including limb and kidney anomalies), failure to thrive, developmental delay and liver disease

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 Lathosterolosis, sourced from HPO and Orphanet clinical annotations.

Horseshoe kidneyGingival overgrowthHigh palateMicrocephalyEpicanthusFull cheeksSloping foreheadNarrow foreheadLong philtrumMicrognathiaHearing impairmentBulbous noseAnteverted naresMicrocorneaDownslanted palpebral fissuresPtosisCataractPostaxial hand polydactylySeizureHypotoniaGlobal developmental delaySpecific learning disabilityMyoclonusHepatic failureIntrahepatic cholestasisFailure to thriveIntrauterine growth retardationToe syndactylyPostaxial foot polydactylyThrombocytopeniaTalipesHepatomegalyChiari malformationMeningoceleCerebral calcificationDownturned corners of mouthShort noseBiparietal narrowingAnisopoikilocytosisProminent metopic ridgeOpacification of the corneal stromaCerebellar cortical atrophyHypoplasia of penisAbnormal platelet morphologyAbnormal thoracic spine morphology

Classification & Codes

MeSH Code

C537880

Orphanet Code

ORPHA:46059
Lathosterolosis
MeSHC537880
OrphanetORPHA:46059
Treatments0 drug(s)
Symptoms on record45 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO