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Gangliosidoses, GM2
A group of recessively inherited diseases characterized by the intralysosomal accumulation of G(M2) GANGLIOSIDE in the neuronal cells. Subtypes include mutations of enzymes in the BETA-N-ACETYLHEXOSAMINIDASES system or G(M2) ACTIVATOR PROTEIN leading to disruption of normal degradation of GANGLIOSIDES, a subclass of ACIDIC GLYCOSPHINGOLIPIDS.
Subtype terms (3)
More specific conditions that are subtypes of Gangliosidoses, GM2 in the MeSH hierarchy.
- Sandhoff Disease
19 drugs (14 approved, 5 experimental)
- Tay-Sachs Disease
19 drugs (14 approved, 5 experimental)
- Tay-Sachs Disease, AB Variant
Drugs by status
Phase 4 trials (1)
Phase 2 trials (4)
Organizations
Organization Involved with Phase 4 Indications (6)
Organization Involved with Phase 3 Indications (5)
Organization Involved with Phase 2 Indications (2)
Organization Involved with Phase 1 Indications (3)