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Substrate reduction therapy in the infantile form of Tay-Sachs disease.
Neurology. 2006 Jan 24;66(2):278-80. doi: 10.1212/01.wnl.0000194225.78917.de.
Neurology. 2006.
PMID: 16434676
Treatment with miglustat reverses the lipid-trafficking defect in Niemann-Pick disease type C.
Lachmann RH, te Vruchte D, Lloyd-Evans E, Reinkensmeier G, Sillence DJ, Fernandez-Guillen L, Dwek RA, Butters TD, Cox TM, Platt FM.
Lachmann RH, et al. Among authors: fernandez guillen l.
Neurobiol Dis. 2004 Aug;16(3):654-8. doi: 10.1016/j.nbd.2004.05.002.
Neurobiol Dis. 2004.
PMID: 15262277
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