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The mode of action in preventing and treating adrenoleukodystrophy (ALD) primarily involves addressing the underlying biochemical defect caused by mutations in the ABCD1 gene, which impairs the transport of very long-chain fatty acids (VLCFAs) into peroxisomes for degradation. Treatments such as Lorenzo's Oil (a mix of oleic and erucic acids) aim to reduce VLCFA accumulation by altering fatty acid metabolism. Hematopoietic stem cell transplantation can also be utilized in early-onset ALD to restore normal enzyme function and halt disease progression. Gene therapy is being explored as a potential future treatment to directly correct the genetic defect.

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