Pharmacological complementation remedies an inborn error of lipid metabolism
M. D. Hartley, M. D. Shokat, M. J. DeBell, T. Banerji, L. L. Kirkemo, T. S. Scanlan
This study tests two pharmacological strategies to enhance sobetirome's efficacy in lowering VLCFAs in X-ALD mice, finding that Sob-AM2 improves CNS VLCFA reduction but is limited by turnover kinetics.
bioRxiv · Jan 31, 2020Pharmacology & Toxicology