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Chronic visceral acid sphingomyelinase deficiency

ORPHA:77293· ICD-10 E75.2

Definition

A rare autosomal recessive, chronic, acid sphingomyelinase deficiency characterized clinically by onset in childhood with hepatosplenomegaly, growth retardation, interstitial lung disease and absence of neurodegenerative disorders.

Prevalence
Unknown
Inheritance
Autosomal recessive
Age of onset
Childhood