Pharmacological complementation remedies an inborn error of lipid metabolism

X-linked adrenoleukodystrophy (X-ALD) is a rare, genetic disease in which increased very long chain fatty acids (VLCFAs) in the central nervous system (CNS) cause demyelination and axonopathy, leading to neurological deficits. Sobetirome, a potent thyroid hormone agonist, has been shown to lower VLC...

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Detaylı Bibliyografya
Yayımlandı:Cell Chem Biol
Asıl Yazarlar: Hartley, Meredith D., Shokat, Mitra D., DeBell, Margaret J., Banerji, Tania, Kirkemo, Lisa L., Scanlan, Thomas S.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2020
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Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC7245556/
https://ncbi.nlm.nih.gov/pubmed/32169163
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.chembiol.2020.02.008
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