Diseases [C] » Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] » Genetic Diseases, Inborn » Metabolism, Inborn Errors » Carbohydrate Metabolism, Inborn Errors » Glycogen Storage Disease
Diseases [C] » Nutritional and Metabolic Diseases [C18] » Metabolic Diseases » Metabolism, Inborn Errors » Carbohydrate Metabolism, Inborn Errors » Glycogen Storage Disease
Description
A group of inherited metabolic disorders involving the enzymes responsible for the synthesis and degradation of glycogen. In some patients, prominent liver involvement is presented. In others, more generalized storage of glycogen occurs, sometimes with prominent cardiac involvement. MeSH
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Subtype Terms (9)
Glycogen Storage Disease Type I
7 drugs (4 approved, 3 experimental)
Glycogen Storage Disease Type II
30 drugs (16 approved, 14 experimental)
Glycogen Storage Disease Type IIb
1 experimental drug
Glycogen Storage Disease Type III
5 drugs (1 approved, 4 experimental)
Glycogen Storage Disease Type IV
3 drugs (1 approved, 2 experimental)
Glycogen Storage Disease Type V
5 drugs (2 approved, 3 experimental)
Glycogen Storage Disease Type VI
Glycogen Storage Disease Type VII
2 drugs (1 approved, 1 experimental)
Phase 4 Indicated Drugs (1)
Phase 1 Indicated Drugs (1)
Other Experimental Indicated Drugs (3)
Organization Involved with Phase 2 Indications (12)
Organization Involved with Phase 1 Indications (1)
Organization Involved with Other Experimental Indications (3)
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UMLS Data
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