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Globoid Cell Leukodystrophy (Krabbe Disease) Summary

Description: An autosomal recessive metabolic disorder caused by a deficiency of GALACTOSYLCERAMIDASE leading to intralysosomal accumulation of galactolipids such as GALACTOSYLCERAMIDES and PSYCHOSINE. It is characterized by demyelination associated with large multinucleated globoid cells, predominantly involving the white matter of the central nervous system. The loss of MYELIN disrupts normal conduction of nerve impulses.

Also Known As: Krabbe Disease; Leukodystrophy, Globoid; Krabbe Leukodystrophy; Krabbe's Disease; Leukodystrophy, Krabbe Show All >>

Networked: 416 relevant articles (17 outcomes, 26 trials/studies) for this Disease

Key Drugs and Agents for Globoid Cell Leukodystrophy

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Galactosylceramidase : 4 outcomes 4 studies in 208 results : IBA
  2. Psychosine (Galactosylsphingosine) : 3 outcomes 7 studies in 74 results : IBA
  3. beta-Galactosidase (Lactaid) : 1 outcome 1 study in 38 results : IBA
  4. galactocerebroside : 1 outcome in 27 results : IBA
  5. Galactocerebrosidase deficiency : 1 outcome in 18 results : IBA
  6. Galactose (Galactopyranose) : 1 outcome in 4 results : FDA 3 Generic
  7. Cerebrosides : 1 outcome in 3 results : IBA
  8. Isoflavones : 1 outcome in 1 result : IBA
  9. Dimethyl Sulfoxide (DMSO) : 1 outcome in 1 result : FDA 3 Generic
  10. Antioxidants : 1 outcome in 1 result : IBA
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Diseases Related to Globoid Cell Leukodystrophy

  1. Globoid Cell Leukodystrophy (Krabbe Disease)
  2. Demyelinating Diseases (Demyelinating Disease)
  3. Metachromatic Leukodystrophy (Sulfatide Lipidosis)
  4. Gaucher Disease (Gaucher's Disease)
  5. Adrenoleukodystrophy (Adrenoleukodystrophy, X-Linked)
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Key Therapies for Globoid Cell Leukodystrophy

Efficacy Chart >>
  1. Bone Marrow Transplantation (Transplantation, Bone Marrow) : 3 outcomes in 20 results
  2. Hematopoietic Stem Cell Transplantation : 1 outcome 1 study in 12 results
  3. Cell Transplantation : 1 outcome 1 study in 7 results
  4. Transplants (Transplant) : 1 outcome 1 study in 3 results
  5. Enzyme Replacement Therapy : 1 outcome in 4 results
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