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Showing articles 0 to 6 of 6 Filter Applied: lysosomal storage disease (Click to remove) Intrathecal 2-hydroxypropyl-�-cyclodextrin Decreases Neurological Disease Progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial Lancet 390:1758-1768, Ory, D.S.,et al, 2017 Clinicopathologic Conference, Tay-Sacks Disease (GM2, Gangliosidosis) NEJM 370:1830-1841, Case 14-2014, 2014 Clinicopath Conf, Neuronal Ceroid Lipofuscinosis, Late-Onset Infantile Subtype NEJM 347:672-680, Case 27-2002, 2002 Inborn Errors of Metabolism as a Cause of Neurological Disease in Adults: An Approach to Investigation JNNP 69: 5-12, Gray,R.G.F. et al, 2000 Neuroimaging Findings in Late Infantile GM1 Gangliosidosis AJNR 19:1628-1630, Chen,C-Y.,et al, 1998 Fucosidosis Revisited:A Review of 77 Patients Am J Med Genet 38:111-131, Willems,P.J.,et al, 1991 Showing articles 0 to 6 of 6