LOVASTATIN THERAPY FOR ADRENOLEUKODYSTROPHY

$0M01FY2001RRNIH

Medical University Of South Carolina, Charleston SC

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Abstract

The purpose of this pilot study is to demonstrate the safety and efficacy of lovastatin in lowering the elevated levels of very long chain fatty acids(VLCFA) in patients with X-linked recessive adrenoleukodystrophy (XALD). The demonstration of the safety and efficacy of lovastatin in lowering accumulated VLCFA in vivo as proposed in this pilot study of ALD patients will pave the way for a larger Phase III clinical trial.

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