Wolcott-Rallison syndrome

MeSH: C536739ORPHA: 1667

Overview

Wolcott-Rallison syndrome (WRS) is a very rare genetic disease, characterized by permanent neonatal diabetes mellitus (PNDM) with multiple epiphyseal dysplasia and other clinical manifestations, including recurrent episodes of acute liver failure

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 Wolcott-Rallison syndrome, sourced from HPO and Orphanet clinical annotations.

Neonatal insulin-dependent diabetes mellitusAbnormality of the liverGrowth delaySkeletal dysplasiaShort statureDecreased total neutrophil countMultiple epiphyseal dysplasiaElevated circulating hepatic transaminase concentrationHypoalbuminemiaDecreased body weightAcute hepatic failureMetaphyseal dysplasiaRenal insufficiencyMicrocephalyStrabismusHypothyroidismJaundiceIntellectual disabilitySeizureGlobal developmental delayMuscle weaknessExocrine pancreatic insufficiencyIron deficiency anemiaDehydrationFeverHyperammonemiaKetoacidosisHepatomegalyRecurrent infectionsHyperbilirubinemiaCentral hypothyroidismChronic kidney diseaseIncreased total lymphocyte countBrain imaging abnormalityAscitesAtrial septal defectDouble outlet right ventricleHepatic encephalopathyHyponatremiaGait disturbance

Classification & Codes

MeSH Code

C536739

Orphanet Code

ORPHA:1667
Wolcott-Rallison syndrome
MeSHC536739
OrphanetORPHA:1667
Treatments0 drug(s)
Symptoms on record40 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO