PHENYLBUTYRATE THERAPY FOR ADRENOLEUKODYSTROPHY

$0M01FY2000RRNIH

Johns Hopkins University, Baltimore MD

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Abstract

The specific aims of this study are to determine whether the administration of Lorenzo Oil to neurologically asymptomatic patients with X-linked adrenoleukodystrophy (X-ALD) will diminish frequency of subsequent neurological disability. To determine whether the oral administration of 4-phenylbutyrate (4PBA) ameliorates the abnormality of VLCFA metabolism in X-ALD patients. To bring to completion an on-going study to determine the effects of docosahexaenole (DHA) and arachidonic acid (AA) therapy inpatients with disorders of peroxisome biogenesis.

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