Morquio Syndrome (MPS-IV) Drug: Updates on Treatment Options for Rare Genetic Disorder Morquio Syndr
Morquio syndrome, also known as mucopolysaccharidosis type IV (MPS-IV), is a rare inherited lysosomal storage disease caused by deficiency of the enzyme N-acetylgalactosamine 6-sulfatase (GALNS). This enzyme is responsible for breaking down glycosaminoglycans (GAGs), long chains of sugar molecules important for healthy cellular functioning. Without sufficient GALNS enzyme activity, GAGs accumulate abnormally in tissues and organs throughout the body. Over time, this buildup causes progressive damage and symptoms of the disease.