
N-butyldeoxynojirimycin delays motor deficits, cerebellar microgliosis, and Purkinje cell loss in a mouse model of mucolipidosis type IV
Keywords: NB-DNJ; N-butyldeoxynojirimycin; GCS; glucosylceramide synthase; GSL; glycosphingolipid; CMH; ceramide monohexoside; CDH; ceramide dihexoside; GBA2; glucocerebrosidase; NPC; Niemann-Pick type C; SRT; substrate reduction therapy; Mucolipidosis type IV; Mig