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} }
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
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
Conditions | Niemann-Pick Disease, Type C |
---|---|
Intervention | Drug |
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
Conditions | Niemann-Pick Disease, Type C1 |
---|---|
Intervention | Drug |
131 Citations
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