Glycogen Storage Disease
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.
Subtype terms (9)
More specific conditions that are subtypes of Glycogen Storage Disease in the MeSH hierarchy.
- Glycogen Storage Disease Type I
10 drugs (4 approved, 6 experimental)
- Glycogen Storage Disease Type II
32 drugs (17 approved, 15 experimental)
- Glycogen Storage Disease Type IIb
1 drug experimental
- 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)
- Glycogen Storage Disease Type VIII
Drugs by status
Phase 4 trials (1)
Phase 2 trials (2)
Phase 1 trials (1)
Other trials (3)
Organizations
Organization Involved with Phase 2 Indications (12)
- Spark Therapeutics
- Baylor University
- University of London
- Institut National de la Santé Et de la Recherche Médicale, France
- University of Groningen
- University of California, Irvine
- University of Copenhagen
- Université catholique de Louvain
- Ultragenyx Inc.
- University of Paris
- Orpha Labs
- The University of Texas, Dallas
Organization Involved with Phase 1 Indications (1)
Organization Involved with Other Experimental Indications (3)