Mucopolysaccharidosis type 4A

A rare autosomal recessive lysosomal storage disease caused by deficiency of the enzyme galactosamine-6-sulfatase. It is characterized by skeletal and central nervous system deficits.

Also known as: Morquio A disease, GALNS deficiency, MPS IV A, MPS4A, MPSIVA, Morquio disease type A, Morquio syndrome A, N-acetylgalactosamine-6-sulfate sulfatase deficiency

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