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GM2 Gangliosidoses (GM2 Gangliosidosis) Summary

Description: 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.

Also Known As: GM2 Gangliosidosis; Gangliosidoses GM2; Gangliosidoses, GM2; GM2 Gangliosidose; GM2, Gangliosidoses Show All >>

Networked: 186 relevant articles (4 outcomes, 12 trials/studies) for this Disease

Key Drugs and Agents for GM2 Gangliosidoses

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. miglustat (Zavesca) : 1 outcome 1 study in 4 results : FDA 1
  2. Pyrimethamine (Daraprim) : 1 outcome in 3 results : FDA 2
  3. Levodopa (L Dopa) : 1 outcome in 1 result : FDA 31 Generic
  4. Hexosaminidase A (Hex A) : 3 studies in 47 results : IBA
  5. Hexosaminidases (Hexosaminidase) : 2 studies in 31 results : IBA
  6. G(M2) Ganglioside (Ganglioside GM2) : 1 study in 32 results : IBA
  7. Oligosaccharides : 1 study in 4 results : IBA
  8. Ceroid : 1 study in 3 results : IBA
  9. Messenger RNA (mRNA) : 1 study in 2 results : IBA
  10. A-Form DNA (A-DNA) : 1 study in 1 result : IBA
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Diseases Related to GM2 Gangliosidoses

  1. Hypokinesia (Bradykinesia)
  2. Gaucher Disease (Gaucher's Disease)
  3. Mannosidase Deficiency Diseases (Mannosidosis)
  4. Sandhoff Disease (Sandhoff's Disease)
  5. GM1 Gangliosidosis (Gangliosidosis GM1)
Show All >>

Key Therapies for GM2 Gangliosidoses

Efficacy Chart >>
  1. Investigational Therapies (Experimental Therapy) : 1 outcome in 1 result
  2. Enzyme Therapy : 1 result
  3. Enzyme Replacement Therapy : 1 result
  4. Bone Marrow Transplantation (Transplantation, Bone Marrow) : 1 result
  5. Activator Appliances (Function Activator) : 1 result

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