Novel SPTBN2 gene mutation and first intragenic deletion in early onset spinocerebellar ataxia type 5

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Abstract

In the present study, we describe two novel cases of SCA5 with early onset. The first one, carrying a novel heterozygous de novo missense mutation in SPTBN2 gene, showed a striking very severe cerebellar atrophy and reduction of volume of the pons at a very young age (16 months). The latter, carrying the first de novo intragenic deletion so far reported in SPTBN2 gene, showed a mild cerebellar atrophy involving the hemispheres and a later onset. In both cases, for the first time, a hyperintense signal of the dentate nuclei was observed.

Original languageEnglish
Pages (from-to)956-963
Number of pages8
JournalAnnals of Clinical and Translational Neurology
Volume8
Issue number4
DOIs
Publication statusPublished - 2021

ASJC Scopus subject areas

  • Neuroscience(all)
  • Clinical Neurology

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