SLC35A2-CDG: novel variants with two ends of the spectrum

J Pediatr Endocrinol Metab. 2021 Jun 22;34(9):1185-1189. doi: 10.1515/jpem-2021-0292. Print 2021 Sep 27.

Abstract

Objectives: Congenital disorders of glycosylation (CDGs) are rare inherited metabolic disorders associated with facial dysmorphism and in the majority of the patients, there is an important neurological impairment. Epilepsy was a main concern in rare forms of the disease. There are two groups of the disease: CDG-I results from the defects in glycan addition to the N-terminal and CDG-II occurs due to defects in the processing of protein bound glycans. SLC35A2-CDG is a rare form of CDG caused by mutations in the X-linked gene that encodes a UDP-Galactose transporter. The manifestations of the disease include seizures, failure to thrive, delayed myelination, and cerebral atrophy.

Case presentation: We describe herein a severe female child with intractable seizures, microcephaly, growth retardation, hypotonia, global developmental delay, facial dysmorphism, skeletal findings, cerebral/cerebellar atrophy, and thin corpus callosum, and a mildly affected male carrying a novel variant with seizures and mild global developmental delay who were found by whole exome sequencing (WES) for SLC35A2 mutations previously not reported.

Conclusions: Our findings expand the number of reported cases and add novel variants to the repertoire of SLC35A2-CDG.

Keywords: SLC35A2-CDG; congenital glycosylation disorders; novel variants.

Publication types

  • Case Reports

MeSH terms

  • Abnormalities, Multiple / genetics
  • Abnormalities, Multiple / pathology*
  • Child, Preschool
  • Congenital Disorders of Glycosylation / complications
  • Congenital Disorders of Glycosylation / genetics
  • Congenital Disorders of Glycosylation / pathology*
  • Epilepsy / complications
  • Epilepsy / genetics
  • Epilepsy / pathology*
  • Female
  • Humans
  • Male
  • Monosaccharide Transport Proteins / genetics*
  • Mutation*
  • Prognosis
  • Seizures / complications
  • Seizures / genetics
  • Seizures / pathology*

Substances

  • Monosaccharide Transport Proteins
  • UDP-galactose translocator

Supplementary concepts

  • Congenital disorder of glycosylation type II