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Published on: August 15, 2019
Juvenile/adult-type galactosialidosis with a homozygous CTSA variant without consanguinity
Machiko Toki1, Kazushige Tsunoda2, Tetsumin So3
1Department of Pediatrics, NHO Tokyo Medical Center, Tokyo, Japan.
Abstract:
Here we report a Japanese patient with juvenile/adult-type galactosialidosis carrying a homozygous c.692+3A>G CTSA variant. Comprehensive genetic analyses including exome sequencing, chromosomal microarray and homozygosity mapping supported biallelic inheritance of this variant and suggested a founder effect in the Japanese population. Clinically, the patient exhibited typical features of the juvenile/adult-type galactosialidosis, with growth impairment noted during adolescence as a less conspicuous but relevant observation.
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