Autosomal recessive spastic ataxia of Charlevoix-Saguenay

Last revised by Rohit Sharma on 28 Feb 2024

Autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) is a rare autosomal recessive spastic ataxia initially identified in the region of Charlevoix-Saguenay, in the province of Quebec, Canada.

Although initially described in Canada, ARSACS has since been reported in other regions of the world, including the Netherlands 9, Brazil 7, Italy 4, France 10, Iran 13, Japan, Turkey and Tunisia 12.

ARSACS typically manifests in infancy, however, late-onset (i.e. adult-onset) forms have been described. The classic triad of presentation clinical features includes 13:

  • cerebellar ataxia

  • pyramidal involvement, leading to spasticity

    • preferentially affecting the lower limbs

  • sensorimotor polyneuropathy

Other clinical features include distal amyotrophy, dysarthria, retinal optic nerve hypermyelination, bowel and bladder dysfunction, and intellectual disability 12,13.

CARSACS is caused by mutation in the SACS gene, which is located on chromosome 13q12.12 and is inherited in an autosomal recessive pattern 12. The SACS gene codes for the protein sacsin, which is expressed highly in motor neurons and by cerebellar Purkinje cells 13.

Common radiographic features on MRI include 1-5,11,13,14:

  • atrophy of the superior cerebellar vermis

    • inferior vermis remains relatively spared

  • T2/FLAIR hypointensities in the pons with a characteristic tigroid, linear, striated or striped appearance

  • cervical cord atrophy

  • T2 hyperintense rim around the thalamus

  • corpus callosum thinning

Management is supportive as there is no disease-modifying treatment available.

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