TREATMENT OF PEROXISOME BIOGENESIS DISORDER

摘要

A method is described for treating a peroxisome biogenesis disorder by administering to an individual in need thereof an effective amount of an autophagy inhibitor. Uses, compositions, and commercial packages are also described. The peroxisome biogenesis disorder may be Zellweger syndrome, neonatal adrenoleukodystrophy, Refsum disease, or cerebrohepatorenal syndrome. The autophagy inhibitor may be chloroquine diphosphate; hydroxychloroquine sulfate; verteporfin; difluoromethylornithine; clarithromycin; clomipramine; desmethylclomipramine hydrochloride, anisomycin; Spautin-1; U0126; SP600125; Wortmannin; LY294002; Bafilomycin; Forskolin; Melatonin; 1-((2-(diethylamino)ethyl)amino)-4-methylthioxanthen-9-one; 1-(2-diethylaminoethylamino)-4-(hydroxymethyl)-9-thioxanthenone; or N-[[1-[[2-(diethylamino)ethyl]amino]-9-oxo-9H-thiaxanthen-4-yl]methyl]methanesulfonamide.