Program

2
2

Journal Year Published

1
1
1
1

Author

4
185
178
145
136
125
123
118
117
108
99
88
81
77
58
54
50
46
46
42
37

Category

2
2
1
1

Keyword

1
1
1
1
1
1
1
1
1
1
1
1
1

Journal

1
1
1
1

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

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

Sample image from publication page

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

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