Related MeSH Hierarchy (5)
Diseases [C] » Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] » Genetic Diseases, Inborn » Metabolism, Inborn Errors » Carbohydrate Metabolism, Inborn Errors » Mucopolysaccharidoses
Diseases [C] » Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] » Genetic Diseases, Inborn » Metabolism, Inborn Errors » Lysosomal Storage Diseases » Mucopolysaccharidoses
Diseases [C] » Skin and Connective Tissue Diseases [C17] » Connective Tissue Diseases » Mucinoses » Mucopolysaccharidoses
Diseases [C] » Nutritional and Metabolic Diseases [C18] » Metabolic Diseases » Metabolism, Inborn Errors » Carbohydrate Metabolism, Inborn Errors » Mucopolysaccharidoses
Diseases [C] » Nutritional and Metabolic Diseases [C18] » Metabolic Diseases » Metabolism, Inborn Errors » Lysosomal Storage Diseases » Mucopolysaccharidoses
Description
Group of lysosomal storage diseases each caused by an inherited deficiency of an enzyme involved in the degradation of glycosaminoglycans (mucopolysaccharides). The diseases are progressive and often display a wide spectrum of clinical severity within one enzyme deficiency. MeSH
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Subtype Terms (6)
Mucopolysaccharidosis I
25 drugs (17 approved, 8 experimental)
Mucopolysaccharidosis II
26 drugs (13 approved, 13 experimental)
Mucopolysaccharidosis III
18 drugs (7 approved, 11 experimental)
Mucopolysaccharidosis IV
4 drugs (3 approved, 1 experimental)
Mucopolysaccharidosis VI
18 drugs (12 approved, 6 experimental)
Mucopolysaccharidosis VII
11 drugs (8 approved, 3 experimental)
Phase 4 Indicated Drugs (5)
Phase 3 Indicated Drugs (3)
Phase 1 Indicated Drugs (5)
Other Experimental Indicated Drugs (1)
Organization Involved with Phase 4 Indications (9)
Organization Involved with Phase 3 Indications (60)
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
Federal University of Rio de Janeiro
Justus Liebig University Giessen
Los Angeles Biomedical Research Institute
State University of New York, Syracuse
University Hospital Center of Sao Joao (UHCSJ)
University of California, Irvine
Organization Involved with Phase 2 Indications (19)
Organization Involved with Phase 1 Indications (7)
Organization Involved with Other Experimental Indications (5)
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UMLS Data
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