Therapeutic potential of deuterium‐stabilized (R)‐pioglitazone—PXL065—for X‐linked adrenoleukodystrophy
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DOI: 10.1002/jimd.12510
Abstract: X‐linked adrenoleukodystrophy (ALD) results from ABCD1 gene mutations which impair Very Long Chain Fatty Acids (VLCFA; C26:0 and C24:0) peroxisomal import and β‐oxidation, leading to accumulation in plasma and tissues. Excess VLCFA drives impaired cellular… read more here.
Keywords: pioglitazone pxl065; deuterium stabilized; linked adrenoleukodystrophy; pioglitazone ... See more keywords