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Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C mice.
Nietupski JB, Pacheco JJ, Chuang WL, Maratea K, Li L, Foley J, Ashe KM, Cooper CG, Aerts JM, Copeland DP, Scheule RK, Cheng SH, Marshall J. Nietupski JB, et al. Among authors: chuang wl. Mol Genet Metab. 2012 Apr;105(4):621-8. doi: 10.1016/j.ymgme.2012.01.020. Epub 2012 Feb 1. Mol Genet Metab. 2012. PMID: 22366055
Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency:the utility of animal models of disease in the toxicological evaluation of potential therapeutics.
Murray JM, Thompson AM, Vitsky A, Hawes M, Chuang WL, Pacheco J, Wilson S, McPherson JM, Thurberg BL, Karey KP, Andrews L. Murray JM, et al. Among authors: chuang wl. Mol Genet Metab. 2015 Feb;114(2):217-25. doi: 10.1016/j.ymgme.2014.07.005. Epub 2014 Jul 12. Mol Genet Metab. 2015. PMID: 25092414 Free article.
659 results