What Is Known And Objective: Adrenoleukodystrophy (ALD) is an X-linked disorder and characterized by the accumulation of saturated very long-chain fatty acids. Treatment is still unsatisfactory. Our objective is to report on the effect of the free-radical scavenger, edaravone, in a patient with ALD.

Case Summary: The patient was given edaravone intravenously twice. D-ROM in cerebral spinal fluid decreased dramatically, and a shortening of neuronal transmission time as estimated on somatosensory evoked potential was observed. After terminating the treatment, his symptoms progressively reappeared.

What Is New And Conclusion: This is the first report of the use of edaravone in ALD. The drug is apparently effective in improving symptoms of ALD and should be evaluated more formally.

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http://dx.doi.org/10.1111/j.1365-2710.2011.01267.xDOI Listing

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