Mucopolysaccharidosis type 3A

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.

Also known as: MPS III A, MPS3A, MPSIIIA, Sanfilippo A, Sanfilippo syndrome a, Sanfilippo syndrome type A, heparan sulfamidase deficiency, mucopolysaccharidosis type 3A

8 clinical trials for this condition and its sub-types.