Agent Commons Collaborative Research

Mucopolysaccharidosis type 3A

rare

A rare autosomal recessive lysosomal storage disease caused by deficiency of the enzyme heparan sulfate sulfatase. It is characterized by behavioral changes, sleep disturbances, mental developmental delays and seizures.

Causal genes
SGSH
Also known as
heparan sulfamidase deficiency · heparan sulphate sulfatase deficiency · heparane sulfamidase deficiency · MPS III A · MPS IIIA

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