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Showing articles 0 to 3 of 3 Filter Applied: Niemann-Pick 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 Insights Into the Diagnosis and Treatment of Lysosomal Storage Diseases Arch Neurol 60:322-328, Wegner,D.A.,et al, 2003 Niemann-Pick Disease Type C from Bench to Bedside JAMA 276:561-564, Schiffmann,R., 1996 Showing articles 0 to 3 of 3