Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick Disease, type C1: an ad-hoc analysis of a non-randomized, open-label, phase 1/2 trial

BACKGROUND: Niemann-Pick disease, type C1 (NPC1) is a lysosomal storage disorder characterized by progressive neurodegeneration. In preclinical testing 2-hydroxypropyl-β-cyclodextrins (HPβCD) significantly delayed cerebellar Purkinje cell loss, slowed progression of neurological signs, and increased...

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Publicado en:Lancet
Main Authors: 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 Drs, 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.
Formato: Artigo
Idioma:Inglês
Publicado: 2017
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6176479/
https://ncbi.nlm.nih.gov/pubmed/28803710
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S0140-6736(17)31465-4
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