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NCATS Publications

2-Hydroxypropyl-β-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 disease.


Davidson J, Molitor E, Moores S, Gale SE, Subramanian K, Jiang X, Sidhu R, Kell P, Zhang J, Fujiwara H, Davidson C, Helquist P, Melancon BJ, Grigalunas M, Liu G, Salahi F, Wiest O, Xu X, Porter FD, Pipalia NH, Cruz DL, Holson EB, Schaffer JE, Walkley SU, Maxfield FR, Ory DS


Biochim Biophys Acta Mol Cell Biol Lipids , (1864), 1545-1561, 2019.

Article Pubmed

Sample image from publication page

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