Article
Etiology and treatment of adrenoleukodystrophy: new insights from Drosophila.
Disease models & mechanisms - 15 Jun 2018
Gordon Hannah B, Valdez Lourdes, Letsou Anthea
Abstract excerpt
Adrenoleukodystrophy (ALD) is a fatal progressive neurodegenerative disorder affecting brain white matter. The most common form of ALD is X-linked (X-ALD) and results from mutation of the ABCD1-encoded very-long-chain fatty acid (VLCFA) transporter. X-ALD is clinically heterogeneous, with the cerebral form being the most severe. Diagnosed in boys usually between the ages of 4 and 8 years, cerebral X-ALD symptoms...
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