Article
[Adrenoleukodystrophy: molecular pathogenesis and development of therapeutic agents].
Yakugaku zasshi : Journal of the Pharmaceutical Society of Japan - 1 Jul 2007
Morita Masashi
Abstract excerpt
Adrenoleukodystrophy (ALD) is an inherited disorder characterized by progressive demyelination of the central nervous system and adrenal dysfunction. The biochemical characterization is made based on the accumulation of pathognomonic amounts of saturated very long chain fatty acid (VLCFA, >22) in all tissues, including brain white matter and adrenal glands. The accumulation of VLCFA is linked to a mutation in the...
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