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

@article{Ory2017Intrathecal2D,
  title={Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial},
  author={D. Ory and E. Ottinger and N. Farhat and K. King and X. Jiang and L. Weissfeld and E. Berry-Kravis and C. Davidson and S. Bianconi and Lee Ann Keener and R. Rao and A. Soldatos and R. Sidhu and Kimberly A Walters and X. Xu and A. Thurm and B. Solomon and W. Pavan and Bernardus N Machielse and M. Kao and S. Silber and J. McKew and C. Brewer and C. Vite and S. Walkley and C. Austin and F. Porter},
  journal={The Lancet},
  year={2017},
  volume={390},
  pages={1758-1768}
}
  • D. Ory, E. Ottinger, +24 authors F. Porter
  • Published 2017
  • Medicine
  • The Lancet
  • BACKGROUND 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. METHODS In this open-label, dose-escalation phase 1-2a… CONTINUE READING
    131 Citations

    Paper Mentions

    Interventional Clinical Trial
    Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence. NPC… Expand
    ConditionsNiemann-Pick Disease, Type C
    InterventionDrug
    Interventional Clinical Trial
    Background: For people who have Niemann-Pick disease, type C1 (NPC1), cholesterol and other fats have trouble moving out of liver and other tissue cells. This makes the cells sick… Expand
    ConditionsNiemann-Pick Disease, Type C1
    InterventionDrug
    Use of 2-Hydroxypropyl-Beta-Cyclodextrin for Niemann-Pick Type C Disease
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    2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1
    • P. Calias
    • Medicine
    • Current pharmaceutical design
    • 2017
    • 18
    • Highly Influenced
    • PDF
    2-Hydroxypropyl-β-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 disease.
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