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Morquio Syndrome (MPS-IV) Drug: Updates on Treatment Options for Rare Genetic Disorder Morquio Syndrome
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...
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