PROPHYLACTIC OR THERAPEUTIC COMPOSITION FOR X-LINKED ADRENOLEUKODYSTROPHY

摘要

The present invention relates to an amiloride derivative compound as an efficient therapeutic composition for diseases associated with excessive accumulation of long-chain fatty acids, especially x-linked adrenoleukodystrophy. The composition of the present invention provides a fundamental therapy method for x-linked adrenoleukodystrophy, which is an incurable disease, and the like by significantly increasing an activity of ABCD2 genetic promoter, which has a compensatory effect for ABCD1 protein participating in degradation of long-chain fatty acids, and decreasing the level of C26:0 and C24:0 long-chain fatty acids in a concentration-dependent manner while also lowering significantly the ratio of C26:0/C22:0.