Abstract
X-linked Adrenoleukodystrophy (ALD and adrenomyeloneuropathy - AMN) is a neurologic peroxisomal disorder, caused by ABCD1-gene mutations, leading to Very Long Chain Fatty Acid (VLCFA; C26:0) accumulation, AMPK downregulation, inflammation, mitochondrial impairment and demyelination. We investigated PXL770, a clinical-stage new direct AMPK activator, in AMN/ALD models.
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