Niemann–Pick Disease Type C
A hopeful therapy for Niemann-Pick C diseases - The Lancet
Experimental treatment for Niemann-Pick disease appears safe
Elucidating the mechanism of cyclodextrins in the treatment of Niemann-Pick Disease Type C using crosslinked 2-hydroxypropyl-β-cyclodextrin | bioRxiv
Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann–Pick Type C Using Quantitative Proteomics | Journal of the American Society for Mass Spectrometry
Linear Cyclodextrin Polymer Prodrugs as Novel Therapeutics for Niemann-Pick Type C1 Disorder | Scientific Reports
Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library
IJMS | Free Full-Text | In Vitro and In Vivo Evaluation of 6-O-α-Maltosyl-β- Cyclodextrin as a Potential Therapeutic Agent Against Niemann-Pick Disease Type C
Cyclodextrin for Niemann-Pick Type C1 Disease | National Center for Advancing Translational Sciences
Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrin - ScienceDirect
Niemann–Pick disease - Wikipedia
IJMS | Free Full-Text | Neurodegeneration in Niemann–Pick Type C Disease: An Updated Review on Pharmacological and Non-Pharmacological Approaches to Counteract Brain and Cognitive Impairment
Oxidative Stress: A Pathogenic Mechanism for Niemann-Pick Type C Disease
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial - The Lancet
Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann–Pick Type C Using Quantitative Proteomics | Journal of the American Society for Mass Spectrometry
![Monitoring the itinerary of lysosomal cholesterol in Niemann-Pick Type C1-deficient cells after cyclodextrin treatment[S] - Journal of Lipid Research](https://www.jlr.org/cms/asset/d8b9eae7-ac30-4e96-b1f9-c0c658361a49/gr1.jpg "Monitoring the itinerary of lysosomal cholesterol in Niemann-Pick Type C1-deficient cells after cyclodextrin treatment[S] - Journal of Lipid Research")
Monitoring the itinerary of lysosomal cholesterol in Niemann-Pick Type C1-deficient cells after cyclodextrin treatment[S] - Journal of Lipid Research
2-Hydroxypropyl-gamma-cyclodextrin overcomes NPC1 deficiency by enhancing lysosome-ER association and autophagy | Scientific Reports
A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1 | BMC Biology | Full Text
Cyclodextrin Shows Promise in Niemann-Pick Type C Disease
Why are people fighting over a promising treatment for a fatal childhood disease? | Science | AAAS
Synthesis of the anionic hydroxypropyl-β-cyclodextrin:poly(decamethylenephosphate) polyrotaxane and evaluation of its cholesterol efflux potential in Niemann– Pick C1 cells - Journal of Materials Chemistry B (RSC Publishing)
Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library
A proposed model for the pathogenesis of Niemann-Pick disease type... | Download Scientific Diagram
Pluronic based β-cyclodextrin polyrotaxanes for treatment of Niemann-Pick Type C disease | Scientific Reports
Investigating the Mechanism of Cyclodextrins in the Treatment of Niemann‐ Pick Disease Type C Using Crosslinked 2‐Hydroxypropyl‐β‐cyclodextrin,Small - X-MOL
