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AICA-рибозидурія

ORPHA:250977· ICD-10 E79.8· AICA-ribosiduria

Визначення(English summary)

A rare and severe inborn metabolic disease characterized clinically by the association of severe-to-profound neurodevelopmental impairment, severe visual impairment, ante-postnatal growth impairment, severe scoliosis and, frequently, early-onset epilepsy.

Поширеність
<1 / 1 000 000
Успадкування
Autosomal recessive
Вік початку
Antenatal, Infancy, Neonatal