Related MeSH Hierarchy (8)
Diseases [C] » Nervous System Diseases [C10] » Neurologic Manifestations » Neurobehavioral Manifestations » Intellectual Disability » Mental Retardation, X-Linked » Mucopolysaccharidosis II
Diseases [C] » Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] » Genetic Diseases, Inborn » Genetic Diseases, X-Linked » Mental Retardation, X-Linked » Mucopolysaccharidosis II
Diseases [C] » Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] » Genetic Diseases, Inborn » Heredodegenerative Disorders, Nervous System » Mental Retardation, X-Linked » Mucopolysaccharidosis II
Diseases [C] » Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] » Genetic Diseases, Inborn » Metabolism, Inborn Errors » Carbohydrate Metabolism, Inborn Errors » Mucopolysaccharidoses » Mucopolysaccharidosis II
Diseases [C] » Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] » Genetic Diseases, Inborn » Metabolism, Inborn Errors » Lysosomal Storage Diseases » Mucopolysaccharidoses » Mucopolysaccharidosis II
Diseases [C] » Skin and Connective Tissue Diseases [C17] » Connective Tissue Diseases » Mucinoses » Mucopolysaccharidoses » Mucopolysaccharidosis II
Diseases [C] » Nutritional and Metabolic Diseases [C18] » Metabolic Diseases » Metabolism, Inborn Errors » Carbohydrate Metabolism, Inborn Errors » Mucopolysaccharidoses » Mucopolysaccharidosis II
Diseases [C] » Nutritional and Metabolic Diseases [C18] » Metabolic Diseases » Metabolism, Inborn Errors » Lysosomal Storage Diseases » Mucopolysaccharidoses » Mucopolysaccharidosis II
Description
Systemic lysosomal storage disease marked by progressive physical deterioration and caused by a deficiency of L-sulfoiduronate sulfatase. This disease differs from MUCOPOLYSACCHARIDOSIS I by slower progression, lack of corneal clouding, and X-linked rather than autosomal recessive inheritance. The mild form produces near-normal intelligence and life span. The severe form usually causes death by age 15. MeSH
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Approved Indicated Drugs (2)
Phase 3 Indicated Drugs (1)
Organization Involved with Phase 4 Indications (6)
Organization Involved with Phase 3 Indications (50)
ASO SS Antonio e Biagio e C Arrigo Alessandria, Italy
Badalona Hospital Germans Trias i Pujol
Comprehensive Cancer Centers of Nevada
Federal University of Mato Grosso do Sul
Justus Liebig University Giessen
Los Angeles Biomedical Research Institute
Mid-Illinois Hematology and Oncology Associates
Organization Involved with Phase 2 Indications (5)
Organization Involved with Phase 1 Indications (4)
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UMLS Data
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