Long-term clinical course of a patient with mucopolysaccharidosis type IIIB

• Main Authors: Ja Hye Kim (Author), Yang Hyun Chi (Author), Gu-Hwan Kim (Author), Han-Wook Yoo (Author), Jun Hwa Lee (Author)
• Formato: Libro
• Publicado: Korean Pediatric Society, 2016-11-01T00:00:00Z.
• Subjects: article
• Acceso en liña: Connect to this object online.

Mucopolysaccharidosis type III (MPS III) is a rare genetic disorder caused by lysosomal storage of heparan sulfate. MPS IIIB results from a deficiency in the enzyme alpha-N-acetyl-D-glucosaminidase (NAGLU). Affected patients begin showing behavioral changes, progressive profound mental retardation,...