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Author

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Journal

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NCATS Publications

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


Ory DS, Ottinger E, Farhat NY, King KA, Jiang X, Weissfeld L, Berry-Kravis E, Davidson C, Bianconi S, Keener LA, Rao R, Soldatos A, Sidhu R, Walters KA, Xu X, Thurm A, Solomon B, Pavan WJ, Machielse BN, Kao M, Silber SA, McKew JC, Brewer CC, Vite CH, Walkley SU, Austin C, Porter FD


Lancet , 2017.

Article Pubmed

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


Walkley SU, Davidson C, Jacoby J, Marella PD, Ottinger E, Austin C, Porter FD, Vite CH, Ory DS


Orphanet J Rare Dis , (11), 161, 2016.

Article Pubmed

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δ-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders.


Xu M, Liu K, Swaroop M, Porter FD, Sidhu R, Firnkes S, Finkes S, Ory DS, Marugan J, Xiao J, Southall N, Pavan WJ, Davidson C, Walkley SU, Remaley AT, Baxa U, Sun W, McKew JC, Austin C, Zheng W


J. Biol. Chem. , (287), 39349-60, 2012.

Article Pubmed

Sample image from publication page