acute infantile liver failure-cerebellar ataxia-peripheral sensory motor neuropathy syndrome

ORPHA: 466794

Overview

autosomal recessive cerebellar ataxia that has material basis in homozygous or compound heterozygous mutation in the SCYL1 gene on chromosome 11q13

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 acute infantile liver failure-cerebellar ataxia-peripheral sensory motor neuropathy syndrome, sourced from HPO and Orphanet clinical annotations.

Dysmetric saccadesSaccadic smooth pursuit interruptionsMild intellectual disabilityGlobal developmental delayHyporeflexiaHepatic fibrosisHepatosplenomegalyFeverGait ataxiaProgressive cerebellar ataxiaIntention tremorFrequent fallsParesthesiaSomatic sensory dysfunctionAcute hepatic failureCerebellar vermis atrophyDistal lower limb muscle weaknessGeneralized limb muscle atrophyPeripheral neuropathyStutteringOptic atrophySpasticityHyperreflexiaTalipes equinovarus

Classification & Codes

Orphanet Code

ORPHA:466794
acute infantile liver failure-cerebellar ataxia-peripheral sensory motor neuropathy syndrome
OrphanetORPHA:466794
Treatments0 drug(s)
Symptoms on record24 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO