permanent neonatal diabetes mellitus

ORPHA: 99885

Overview

neonatal diabetes mellitus that has material basis in homozygous mutation in the glucokinase gene (GCK), heterozygous mutation in the KCNJ11 and INS genes, or by heterozygous or homozygous mutation in the ABCC8 gene

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 permanent neonatal diabetes mellitus, sourced from HPO and Orphanet clinical annotations.

Downturned corners of mouthArthrogryposis multiplex congenitaKetonuriaProminent metopic ridgeLower-limb joint contractureModerate albuminuriaNeurodevelopmental delayRenal tubular dysfunctionHearing impairmentAtaxiaHypotoniaComaApraxiaPancreatic hypoplasiaPeripheral axonal neuropathySevere intellectual disabilityAbnormality of the upper urinary tractNeonatal insulin-dependent diabetes mellitusFailure to thriveWeight lossDehydrationHyperglycemiaGlycosuriaReduced pancreatic beta cellsHypovolemiaRetinopathyIntellectual disabilityGlobal developmental delayMotor delayBilateral ptosisIntrauterine growth retardationAbnormal heart morphologyBilateral tonic-clonic seizureGeneralized myoclonic seizure

Classification & Codes

Orphanet Code

ORPHA:99885
permanent neonatal diabetes mellitus
OrphanetORPHA:99885
Treatments0 drug(s)
Symptoms on record34 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO