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      Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial.

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          Abstract

          Niemann-Pick disease, type C1 (NPC1) is a lysosomal storage disorder characterised by progressive neurodegeneration. In preclinical testing, 2-hydroxypropyl-β-cyclodextrins (HPβCD) significantly delayed cerebellar Purkinje cell loss, slowed progression of neurological manifestations, and increased lifespan in mouse and cat models of NPC1. The aim of this study was to assess the safety and efficacy of lumbar intrathecal HPβCD.

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          Author and article information

          Journal
          Lancet
          Lancet (London, England)
          Elsevier BV
          1474-547X
          0140-6736
          Oct 14 2017
          : 390
          : 10104
          Affiliations
          [1 ] Washington University School of Medicine, St Louis, MO, USA.
          [2 ] National Center for Advancing Translational Sciences, National Institutes of Health, Department of Health and Human Services, Bethesda, MD, USA.
          [3 ] Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Health and Human Services, Bethesda, MD USA.
          [4 ] National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Department of Health and Human Services, Bethesda, MD, USA.
          [5 ] Statistics Collaborative, Washington, DC, USA.
          [6 ] Rush University Medical Center, Chicago, IL, USA.
          [7 ] Albert Einstein College of Medicine, Bronx, NY, USA.
          [8 ] Vtesse Inc, Gaithersburg, MD, USA.
          [9 ] National Institute of Neurological Disorders and Stroke, National Institutes of Health, Department of Health and Human Services Bethesda, MD, USA.
          [10 ] National Institute of Mental Health, National Institutes of Health, Department of Health and Human Services, Bethesda, MD, USA.
          [11 ] Mark O Hatfield Clinical Research Center, National Institutes of Health, Department of Health and Human Services, Bethesda, MD, USA.
          [12 ] National Human Genome Research Institute, National Institutes of Health, Department of Health and Human Services, Bethesda, MD, USA.
          [13 ] Preclinical Development and Safety, Janssen R&D, Raritan, NJ, USA.
          [14 ] Global Public Health, Johnson & Johnson, Philadelphia, PA, USA.
          [15 ] School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
          [16 ] Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Health and Human Services, Bethesda, MD USA. Electronic address: fdporter@mail.nih.gov.
          Article
          S0140-6736(17)31465-4
          10.1016/S0140-6736(17)31465-4
          28803710

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