Gaucher's disease perinatal lethal

ORPHA: 85212

Overview

Gaucher's Disease characterized by perinatal lethality and rapid progression of neurological deterioration that has material basis in an autosomal recessive mutation of GBA on chromosome 1q22

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 Gaucher's disease perinatal lethal, sourced from HPO and Orphanet clinical annotations.

IchthyosisHigh palateAnteverted naresEctropionHypotoniaFlexion contractureHepatosplenomegalyFailure to thriveThrombocytopeniaAnemiaConjugated hyperbilirubinemiaDecreased beta-glucocerebrosidase levelDepressed nasal bridgeElevated circulating aspartate aminotransferase concentrationHydrocephalusSeizureHypertoniaNonimmune hydrops fetalisGastroesophageal refluxPoor suckIntracranial hemorrhageOpisthotonusCorneal opacityDecreased fetal movementPancytopeniaArthrogryposis multiplex congenitaCongenital nonbullous ichthyosiform erythrodermaPosteriorly rotated ears

Classification & Codes

Orphanet Code

ORPHA:85212
Gaucher's disease perinatal lethal
OrphanetORPHA:85212
Treatments0 drug(s)
Symptoms on record28 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO