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Gangliosidoses
A group of autosomal recessive lysosomal storage disorders marked by the accumulation of GANGLIOSIDES. They are caused by impaired enzymes or defective cofactors required for normal ganglioside degradation in the LYSOSOMES. Gangliosidoses are classified by the specific ganglioside accumulated in the defective degradation pathway.
Subtype terms (2)
More specific conditions that are subtypes of Gangliosidoses in the MeSH hierarchy.
- Gangliosidoses, GM2
5 drugs (4 approved, 1 experimental)
- Gangliosidosis, GM1
15 drugs (9 approved, 6 experimental)
Drugs by status
Phase 2 trials (1)
Organizations
Organization Involved with Phase 4 Indications (6)
Organization Involved with Phase 3 Indications (6)
Organization Involved with Phase 2 Indications (5)
Organization Involved with Phase 1 Indications (3)