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ArticlesPeer ReviewedFull Text
Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression
Author / Creator:Davidson, Cristin D ; Ali, Nafeeza F ; Micsenyi, Matthew C ; Stephney, Gloria ; Renault, Sophie ; Dobrenis, Kostantin ; Ory, Daniel S ; Vanier, Marie T ; Walkley, Steven U
Publisher:United States: Public Library of Science
Journal title:PloS one, 2009-09, Vol.4 (9), p.e6951
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ArticlesPeer ReviewedFull Text
Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood–brain barrier permeability
Author / Creator:Pontikis, Charles C. ; Davidson, Cristin D. ; Walkley, Steven U. ; Platt, Frances M. ; Begley, David J.
Publisher:Dordrecht: Springer Netherlands
Journal title:Journal of inherited metabolic disease, 2013-05, Vol.36 (3), p.491-498
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ArticlesPeer ReviewedFull Text
Phenotype assessment for neurodegenerative murine models with ataxia and application to Niemann-Pick disease, type C1
Author / Creator:Yerger, Julia ; Cougnoux, Antony C ; Abbott, Craig B ; Luke, Rachel ; Clark, Tannia S ; Cawley, Niamh X ; Porter, Forbes D ; Davidson, Cristin D
Publisher:England: The Company of Biologists Ltd
Journal title:Biology open, 2022-04, Vol.11 (4)
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ArticlesPeer ReviewedFull Text
Efficacy and ototoxicity of different cyclodextrins in Niemann–Pick C disease
Author / Creator:Davidson, Cristin D. ; Fishman, Yonatan I. ; Puskás, István ; Szemán, Julianna ; Sohajda, Tamás ; McCauliff, Leslie A. ; Sikora, Jakub ; Storch, Judith ; Vanier, Marie T. ; Szente, Lajos ; Walkley, Steven U. ; Dobrenis, Kostantin
Publisher:United States: John Wiley & Sons, Inc
Journal title:Annals of clinical and translational neurology, 2016-05, Vol.3 (5), p.366-380
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ArticlesPeer ReviewedFull Text
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 / 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 ; Porter, Forbes D
Publisher:England: Elsevier Ltd
Journal title:The Lancet (British edition), 2017-10, Vol.390 (10104), p.1758-1768
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ArticlesPeer ReviewedFull Text
Standard-flow LC and thermal focusing ESI elucidates altered liver proteins in late stage Niemann–Pick, type C1 disease
Author / Creator:Pergande, Melissa R ; Zarate, Estefania ; Haney-Ball, Carol ; Davidson, Cristin D ; Scesa, Giuseppe ; Givogri, Maria I ; Bongarzone, Ernesto R ; Cologna, Stephanie M
Publisher:London: Newlands Press Ltd
Journal title:Bioanalysis, 2019-06, Vol.11 (11), p.1067-1083
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ArticlesPeer ReviewedFull Text
Fostering collaborative research for rare genetic disease: the example of niemann-pick type C disease
Author / Creator:Walkley, Steven U ; Davidson, Cristin D ; Jacoby, Jonathan ; Marella, Philip D ; Ottinger, Elizabeth A ; Austin, Christopher P ; Porter, Forbes D ; Vite, Charles H ; Ory, Daniel S
Publisher:England: BioMed Central Ltd
Journal title:Orphanet journal of rare diseases, 2016-12, Vol.11 (1), p.161, Article 161
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ArticlesPeer ReviewedFull Text
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
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 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.
Journal title:The Lancet (British edition), 2017-08, Vol.390 (10104), p.1758-1768
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Conference ProceedingsPeer ReviewedFull Text
Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood-brain barrier permeability : Brains4Brain
Author / Creator:PONTIKIS, Charles C ; DAVIDSON, Cristin D ; WALKLEY, Steven U ; PLATT, Frances M ; BEGLEY, David J
Publisher:Heidelberg: Springer
Journal title:Journal of inherited metabolic disease, 2013, Vol.36 (3), p.491-498
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ReportsFull Text
Fostering collaborative research for rare genetic disease: the example of niemann-pick type C disease
Author / Creator:Walkley, Steven U ; Davidson, Cristin D ; Jacoby, Jonathan ; Marella, Philip D ; Ottinger, Elizabeth A ; Austin, Christopher P ; Porter, Forbes D ; Vite, Charles H ; Ory, Daniel S
Publisher:BioMed Central Ltd
Journal title:Orphanet Journal of Rare Diseases, 2016, Vol.11 (1)
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Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression

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Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood–brain barrier permeability

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Phenotype assessment for neurodegenerative murine models with ataxia and application to Niemann-Pick disease, type C1

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Efficacy and ototoxicity of different cyclodextrins in Niemann–Pick C disease

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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

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Standard-flow LC and thermal focusing ESI elucidates altered liver proteins in late stage Niemann–Pick, type C1 disease

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Fostering collaborative research for rare genetic disease: the example of niemann-pick type C disease

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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

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Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood-brain barrier permeability : Brains4Brain

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Fostering collaborative research for rare genetic disease: the example of niemann-pick type C disease

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