• DOI:10.1016/S0140-6736(17)31465-4
Corpus ID: 8841225

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

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