Adrenoleukodystrophy
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).
Drugs by status
Approved for this indication (3)
Phase 4 trials (1)
Phase 3 trials (10)
Phase 2 trials (19)
Phase 1 trials (2)
Organizations
Organization Involved with Phase 3 Indications (13)
- Travere
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- bluebird bio
- National Institutes of Health (NIH)
- Onofre, Aurora Pujol, M.D.
- University of London
- Harvard University
- University of California, Los Angeles
- University of Cincinnati
- Food and Drug Administration
- University of Minnesota
- MedDay Pharmaceuticals
- Minoryx Therapeutics, S.L.
Organization Involved with Phase 2 Indications (15)
- NeuroVia, Inc.
- National Institute of Neurological Disorders and Stroke (NINDS)
- Duke University
- Guangzhou Medical University
- Stanford University
- University of Florida
- University of Louisville
- The University of Hong Kong
- ALD Connect, Inc.
- Shenzhen University
- Fundacion Hesperia
- China Medical University
- Government of Spain
- Spanish Association of Amyotrophic Lateral Sclerosis (adEla)
- Universidad Carlos III Madrid
Organization Involved with Phase 1 Indications (3)
Organization Involved with Other Experimental Indications (2)