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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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| Veröffentlicht in: | Cell Chem Biol |
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| Hauptverfasser: | , , , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
2020
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| Schlagworte: | |
| Online Zugang: | 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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