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Хвороба Тея-Сакса

ORPHA:845· ICD-10 E75.0· Tay-Sachs disease

Визначення(English summary)

A rare autosomal recessive lysosomal disease characterized by accumulation of GM2 gangliosides in the nervous system due to hexosaminidase A deficiency as a consequence of biallelic pathogenic variants in the HEXA gene.

Поширеність
1-9 / 1 000 000
Успадкування
Autosomal recessive
Вік початку
All ages