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Showing articles 0 to 8 of 8 Filter Applied: hepatosplenomegaly (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 Wilson Disease Yamada Textbook of Gastroenterology Chp 102, Metabolic Diseases of Liver, 6th Ed, Sunderam, S.S., & Sokol, R.J., 2016 Clinical Manifestations, Pathologic Features, and Diagnosis of Langerhans Cell Histiocytosis UptoDate Oct. 2014, McClain, K.L., 2014 Inherited Metabolic Diseases of the Nervous System, Hepatolenticular Degeneration (Wilson Disease) Adams & Victors Principles of Neurology, Chp 37, pg 982, Ropper, A.H.,et al, 2014 Niemann-Pick Disease Type C from Bench to Bedside JAMA 276:561-564, Schiffmann,R., 1996 Wilson Disease:Clinical Presentation, Treatment, and Survival Ann Int Med 115:720-726, Stremmel,W.,et al, 1991 Fucosidosis Revisited:A Review of 77 Patients Am J Med Genet 38:111-131, Willems,P.J.,et al, 1991 The Natural History of SLE by Prospective Analysis Medicine 50:85, Estes,D.,et al, 1971 Showing articles 0 to 8 of 8