Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pic...
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial
About this item
Full title
Author / Creator
Ory, Daniel S , Ottinger, Elizabeth A , Farhat, Nicole Yanjanin , King, Kelly A , Jiang, Xuntian , Weissfeld, Lisa , Berry-Kravis, Elizabeth , Davidson, Cristin D , Bianconi, Simona , Keener, Lee Ann , Rao, Ravichandran , Soldatos, Ariane , Sidhu, Rohini , Walters, Kimberly A , Xu, Xin , Thurm, Audrey , Solomon, Beth , Pavan, William J , Machielse, Bernardus N , Kao, Mark , Silber, Steven A , McKew, John C , Brewer, Carmen C , Vite, Charles H , Walkley, Steven U , Austin, Christopher P and Porter, Forbes D
Publisher
England: Elsevier Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: Elsevier Ltd
Subjects
More information
Scope and Contents
Contents
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 o...
Alternative Titles
Full 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
Authors, Artists and Contributors
Author / Creator
Ottinger, Elizabeth A
Farhat, Nicole Yanjanin
King, Kelly A
Jiang, Xuntian
Weissfeld, Lisa
Berry-Kravis, Elizabeth
Davidson, Cristin D
Bianconi, Simona
Keener, Lee Ann
Rao, Ravichandran
Soldatos, Ariane
Sidhu, Rohini
Walters, Kimberly A
Xu, Xin
Thurm, Audrey
Solomon, Beth
Pavan, William J
Machielse, Bernardus N
Kao, Mark
Silber, Steven A
McKew, John C
Brewer, Carmen C
Vite, Charles H
Walkley, Steven U
Austin, Christopher P
Porter, Forbes D
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_1928783738
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_1928783738
Other Identifiers
ISSN
0140-6736,1474-547X
E-ISSN
1474-547X
DOI
10.1016/S0140-6736(17)31465-4
