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Metachromatic Leukodystrophy (Sulfatide Lipidosis) Summary

Description: An autosomal recessive metabolic disease caused by a deficiency of CEREBROSIDE-SULFATASE leading to intralysosomal accumulation of cerebroside sulfate (SULFOGLYCOSPHINGOLIPIDS) in the nervous system and other organs. Pathological features include diffuse demyelination, and metachromatically-staining granules in many cell types such as the GLIAL CELLS. There are several allelic and nonallelic forms with a variety of neurological symptoms.

Also Known As: Sulfatide Lipidosis; Leukodystrophy, Metachromatic; Arylsulfatase A Deficiency; Cerebral sclerosis, Diffuse, Metachromatic Form; Cerebroside Sulfatase Deficiency Show All >>

Networked: 428 relevant articles (15 outcomes, 24 trials/studies) for this Disease

Key Drugs and Agents for Metachromatic Leukodystrophy

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Cerebroside-Sulfatase (Arylsulfatase A) : 4 outcomes 8 studies in 248 results : IBA
  2. Lysergic Acid Diethylamide (LSD) : 2 outcomes in 7 results : IBA
  3. Sulfoglycosphingolipids (Sulfatides) : 1 outcome 3 studies in 37 results : IBA
  4. Saposins : 1 outcome in 19 results : IBA
  5. DNA (Deoxyribonucleic Acid) : 1 outcome in 6 results : IBA
  6. Galactose (Galactopyranose) : 1 outcome in 2 results : FDA 3 Generic
  7. Complementary DNA (cDNA) : 1 outcome in 1 result : IBA
  8. Sphingosine : 1 outcome in 1 result : IBA
  9. Complement System Proteins (Complement) : 1 outcome in 1 result : IBA
  10. Arylsulfatases : 2 studies in 11 results : IBA
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Diseases Related to Metachromatic Leukodystrophy

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

Efficacy Chart >>
  1. Bone Marrow Transplantation (Transplantation, Bone Marrow) : 2 outcomes 2 studies in 24 results
  2. Hematopoietic Stem Cell Transplantation : 2 outcomes in 6 results
  3. Enzyme Replacement Therapy : 1 outcome in 3 results
  4. Immunomodulation : 1 outcome in 1 result
  5. Drug Therapy (Chemotherapy) : 1 outcome in 1 result
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