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={Daniel S Ory and Elizabeth A Ottinger and Nicole Yanjanin Farhat and Kelly A. King and Xuntian Jiang and Lisa Weissfeld and Elizabeth Berry-Kravis and Cristin D. Davidson and Simona Bianconi and Lee Ann Keener and Ravichandran Rao and Ariane Soldatos and Rohini Sidhu and Kimberly A Walters and Xin Xu and Audrey Thurm and Beth Diane Solomon and William J. Pavan and Bernardus N Machielse and Mark Kao and Steven A Silber and John C. McKew and Carmen Brewer and Charles H Vite and Steven U Walkley and Christopher P. Austin and Forbes D Porter},
  journal={The Lancet},
  year={2017},
  volume={390},
  pages={1758-1768}
}
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

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