| 1 |
34296265 | Transcriptome of HPβCD-treated Niemann-Pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics. | 2021 Nov 30 |
2 |
| 2 |
34592985 | A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1. | 2021 Oct 1 |
1 |
| 3 |
32890578 | Delineation of metabolic responses of Npc1-/-nih mice lacking the cholesterol-esterifying enzyme SOAT2 to acute treatment with 2-hydroxypropyl-β-cyclodextrin. | 2020 Dec |
1 |
| 4 |
29390035 | Correction: Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1. | 2018 |
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| 5 |
29617956 | Microglia activation in Niemann-Pick disease, type C1 is amendable to therapeutic intervention. | 2018 Jun 15 |
2 |
| 6 |
28155026 | Early experience with compassionate use of 2 hydroxypropyl-beta-cyclodextrin for Niemann-Pick type C disease: review of initial published cases. | 2017 May |
4 |
| 7 |
28179943 | Synthesis of multi-lactose-appended β-cyclodextrin and its cholesterol-lowering effects in Niemann-Pick type C disease-like HepG2 cells. | 2017 |
3 |
| 8 |
28414792 | Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1. | 2017 |
3 |
| 9 |
28803710 | Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial. | 2017 Oct 14 |
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| 10 |
27572704 | PEG-lipid micelles enable cholesterol efflux in Niemann-Pick Type C1 disease-based lysosomal storage disorder. | 2016 Aug 30 |
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| 11 |
26392920 | Cyclodextrin Alters GABAergic Input to CA1 Pyramidal Cells in Wild-Type But Not in NPC1-Deficient Mice. | 2015 |
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| 12 |
26664628 | Cholesterol lowering effects of mono-lactose-appended β-cyclodextrin in Niemann-Pick type C disease-like HepG2 cells. | 2015 |
3 |
| 13 |
24283970 | Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease. | 2014 |
4 |
| 14 |
24868096 | Development and validation of sensitive LC-MS/MS assays for quantification of HP-β-CD in human plasma and CSF. | 2014 Jul |
1 |
| 15 |
24907126 | Niemann-Pick Disease Type C: Induced Pluripotent Stem Cell-Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy. | 2014 Sep |
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| 16 |
24964810 | Cholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann-Pick C1 (NPC1). | 2014 Nov 15 |
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| 17 |
23881911 | Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling. | 2013 Oct |
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| 18 |
19884502 | Cyclodextrin overcomes deficient lysosome-to-endoplasmic reticulum transport of cholesterol in Niemann-Pick type C cells. | 2009 Nov 17 |
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