autosomal dominant hyperinsulinism due to SUR1 deficiency

ORPHA: 2765751 Treatment Available

Available Treatments (1)

DrugFormStatusCountriesLead Time
diazoxide
Orphan
Oral suspension, 50mg/mLFDA Approved, EMA Approved614d

Clinical Presentation

Signs and symptoms associated with autosomal dominant hyperinsulinism due to SUR1 deficiency, sourced from HPO and Orphanet clinical annotations.

Hyperinsulinemic hypoglycemiaHyperinsulinemiaHypoketotic hypoglycemiaFasting hypoglycemiaIncreased C-peptide levelExcessive insulin response to glucagon testDecreased circulating free fatty acid levelAgitationPallorEpisodic hyperhidrosisLarge for gestational ageTachycardiaPalpitationsHypoglycemic seizuresDrowsinessNeurodevelopmental abnormalityFocal pancreatic islet hyperplasiaDiffuse pancreatic islet hyperplasiaLethargySyncopeHypoglycemic comaHypertrophic cardiomyopathyStatus epilepticusHepatomegalyPolyphagiaLoss of consciousnessFeeding difficultiesType I diabetes mellitus

Classification & Codes

Orphanet Code

ORPHA:276575
autosomal dominant hyperinsulinism due to SUR1 deficiency
OrphanetORPHA:276575
Treatments1 drug(s)
Symptoms on record28 signs
Statuspublished

Treatment Summary

Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO