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Adrenoleukodystrophy (Adrenoleukodystrophy, X-Linked) Summary

Description: An X-linked recessive disorder characterized by the accumulation of saturated very long chain fatty acids in the LYSOSOMES of ADRENAL CORTEX and the white matter of CENTRAL NERVOUS SYSTEM. This disease occurs almost exclusively in the males. Clinical features include the childhood onset of ATAXIA; NEUROBEHAVIORAL MANIFESTATIONS; HYPERPIGMENTATION; ADRENAL INSUFFICIENCY; SEIZURES; MUSCLE SPASTICITY; and DEMENTIA. The slowly progressive adult form is called adrenomyeloneuropathy. The defective gene ABCD1 is located at Xq28, and encodes the adrenoleukodystrophy protein (ATP-BINDING CASSETTE TRANSPORTERS).

Also Known As: Adrenoleukodystrophy, X-Linked; Adrenomyeloneuropathy; X-Linked Adrenoleukodystrophy; ALD (Adrenoleukodystrophy); ALDs (Adrenoleukodystrophy) Show All >>

Networked: 758 relevant articles (15 outcomes, 36 trials/studies) for this Disease

Key Drugs and Agents for Adrenoleukodystrophy

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. erucic acid : 2 outcomes in 17 results : IBA
  2. Fatty Acids (Saturated Fatty Acids) : 1 outcome 5 studies in 276 results : IBA
  3. ATP-Binding Cassette Transporters (ABC Transporters) : 1 outcome 2 studies in 26 results : IBA
  4. Lysophosphatidylcholines (Lysophosphatidylcholine) : 1 outcome in 4 results : IBA
  5. Baclofen : 1 outcome in 2 results : FDA 31 Generic
  6. Severe combined immunodeficiency due to adenosine deaminase deficiency : 1 outcome in 2 results : IBA
  7. Glycosphingolipids : 1 outcome in 1 result : IBA
  8. DNA Polymerase III (Pol III) : 1 outcome in 1 result : IBA
  9. Lorenzo's oil : 4 studies in 34 results : IBA
  10. Enzymes : 3 studies in 8 results : IBA
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Diseases Related to Adrenoleukodystrophy

  1. Metachromatic Leukodystrophy (Sulfatide Lipidosis)
  2. X-Linked Combined Immunodeficiency Diseases (X Linked Severe Combined Immunodeficiency)
  3. Globoid Cell Leukodystrophy (Krabbe Disease)
  4. Neurodegenerative Diseases (Neurodegenerative Disease)
  5. Addison Disease (Addison's Disease)
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Key Therapies for Adrenoleukodystrophy

Efficacy Chart >>
  1. Bone Marrow Transplantation (Transplantation, Bone Marrow) : 2 outcomes 2 studies in 29 results
  2. Stem Cell Transplantation : 1 outcome 2 studies in 6 results
  3. Heterologous Transplantation (Xenotransplantation) : 1 outcome 1 study in 1 result
  4. Hematopoietic Stem Cell Transplantation : 1 outcome in 13 results
  5. Transplantation (Transplant Recipients) : 1 outcome in 12 results
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