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Updated: Jun 3, 2026
An In Vitro Model for the Study of Cellular Pathophysiology in Globoid Cell Leukodystrophy
Published on: October 21, 2014
New treatment of free-radical scavenger in adrenoleukodystrophy
H Kawashima1, S Nishimata, C Ishii
1Department of Paediatrics, Tokyo Medical University, Tokyo, Japan. hisashi@tokyo-med.ac.jp
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.
