severe neurodegenerative syndrome with lipodystrophy

ORPHA: 363400

Overview

human 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 severe neurodegenerative syndrome with lipodystrophy, sourced from HPO and Orphanet clinical annotations.

Progressive encephalopathyHyperinsulinemiaInsulin resistanceReduced subcutaneous adipose tissueProgressive psychomotor deteriorationGeneralized lipodystrophyReduced intraabdominal adipose tissueCognitive impairmentCoarse facial featuresDelayed speech and language developmentSeizureAtaxiaSpasticityMyoclonusTremorHyperreflexiaBrisk reflexesHepatic steatosisHypertriglyceridemiaIncoordinationSleep disturbanceAbnormal pyramidal signHyperactivityHypertensionAcanthosis nigricansCirrhosisCerebral atrophyGait ataxiaGeneralized hirsutismHepatomegalyTetraparesisCaudate atrophyLimb dystoniaNeuronal loss in central nervous systemRespiratory failureMyopathyStatus epilepticus

Classification & Codes

Orphanet Code

ORPHA:363400
severe neurodegenerative syndrome with lipodystrophy
OrphanetORPHA:363400
Treatments0 drug(s)
Symptoms on record37 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO