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Adrenoleukodystrophy (ALD/ X-ALD) is a rare, X-linked disorder secondary to a mutation in the ABCD1 gene, which encodes for a peroxisomal membrane protein, resulting in an excessive accumulation of very-long-chain fatty acids (VLCFAs) in the brain and adrenal glands. A novel, brain penetrant, orally bioavailable and selective PPAR?
In addition, about 40 percent of these cases involve mutations in the ESR1 gene. Current treatments include hormonal therapies that focus on blocking the function of the estrogen receptor, curbing its production or degrading the receptor itself. Orserdu is available in the form of 345 mg tablets to be taken once a day with food.
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