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Sandhoff Disease (Sandhoff's Disease) Summary

Description: An autosomal recessive neurodegenerative disorder characterized by an accumulation of G(M2) GANGLIOSIDE in neurons and other tissues. It is caused by mutation in the common beta subunit of HEXOSAMINIDASE A and HEXOSAMINIDASE B. Thus this disease is also known as the O variant since both hexosaminidase A and B are missing. Clinically, it is indistinguishable from TAY-SACHS DISEASE.

Also Known As: Sandhoff's Disease; Adult Sandhoff Disease; Deficiency Disease, Hexosaminidase A and B; GM2 Gangliosidosis, Type 2; GM2 Gangliosidosis, Type II Show All >>

Networked: 225 relevant articles (5 outcomes, 15 trials/studies) for this Disease

Key Drugs and Agents for Sandhoff Disease

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. N-butyldeoxygalactonojirimycin : 1 outcome 1 study in 2 results : IBA
  2. Biological Markers (Surrogate Marker) : 1 outcome in 1 result : IBA
  3. Tumor Necrosis Factor-alpha (Tumor Necrosis Factor) : 1 outcome in 1 result : IBA
  4. Liposomes (Liposome) : 1 outcome in 1 result : IBA
  5. miglustat (Zavesca) : 4 studies in 10 results : FDA 1
  6. beta-N-Acetylhexosaminidases : 2 studies in 66 results : IBA
  7. Hexosaminidase A (Hex A) : 2 studies in 38 results : IBA
  8. First Step : 2 studies in 2 results : IBA
  9. Hexosaminidases (Hexosaminidase) : 1 study in 57 results : IBA
  10. G(M2) Ganglioside (Ganglioside GM2) : 1 study in 43 results : IBA
Show All >>

Diseases Related to Sandhoff Disease

  1. Lysosomal Storage Diseases (Lysosomal Storage Disease)
  2. Neurologic Manifestations (Neurological Manifestations)
  3. Disease Progression
  4. Tay-Sachs Disease
  5. GM2 Gangliosidoses (GM2 Gangliosidosis)
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Key Therapies for Sandhoff Disease

Efficacy Chart >>
  1. Bone Marrow Transplantation (Transplantation, Bone Marrow) : 1 outcome in 3 results
  2. Caloric Restriction : 2 studies in 3 results
  3. Transplantation (Transplant Recipients) : 1 study in 3 results
  4. Ketogenic Diet : 2 results
  5. Enzyme Replacement Therapy : 1 result
Show All >>

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