Program

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Journal Year Published

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Author

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Category

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Keyword

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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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Safety, pharmacokinetics and sialic acid production after oral administration of N-acetylmannosamine (ManNAc) to subjects with GNE myopathy.


Xu X, Wang A, Latham LL, Celeste F, Ciccone C, Malicdan MC, Goldspiel B, Terse P, Cradock J, Yang N, Yorke S, McKew JC, Gahl WA, Huizing M, Carrillo N


Mol. Genet. Metab. , (122), 126-134, 2017.

Article Pubmed
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Development of an Aryloxazole Class of Hepatitis C Virus Inhibitors Targeting the Entry Stage of the Viral Replication Cycle.


He S, Li K, Lin B, Hu Z, Xiao J, Hu X, Wang A, Xu X, Ferrer-Alegre M, Southall N, Zheng W, Aubé J, Schoenen FJ, Marugan J, Liang TJ, Frankowski KJ


J. Med. Chem. , (60), 6364-6383, 2017.

Article Pubmed
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The phosphatidylinositol-3-phosphate 5-kinase inhibitor apilimod blocks filoviral entry and infection.


Nelson EA, Dyall J, Hoenen T, Barnes AB, Zhou H, Liang JY, Michelotti J, Dewey WH, DeWald LE, Bennett RS, Morris P, Guha R, Klumpp-Thomas C, McKnight C, Chen Y, Xu X, Wang A, Hughes E, Martin S, Thomas C, Jahrling PB, Hensley LE, Olinger GG, White JM


PLoS Negl Trop Dis , (11), e0005540, 2017.

Article Pubmed
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SU9516 Increases α7β1 Integrin and Ameliorates Disease Progression in the mdx Mouse Model of Duchenne Muscular Dystrophy.


Sarathy A, Wuebbles RD, Fontelonga TM, Tarchione AR, Mathews Griner LA, Heredia DJ, Nunes AM, Duan S, Brewer PD, Van Ry T, Hennig GW, Gould TW, Dulcey AE, Wang A, Xu X, Chen C, Hu X, Zheng W, Southall N, Ferrer-Alegre M, Marugan J, Burkin DJ


Mol. Ther. , 2017.

Article Pubmed
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Highly predictive and interpretable models for PAMPA permeability.


Sun H, Nguyen K, Kerns E, Yan Z, Yu KR, Shah P, Jadhav A, Xu X


Bioorg. Med. Chem. , (25), 1266-1276, 2017.

Article Pubmed
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ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice.


Abera MB, Xiao J, Nofziger J, Titus S, Southall N, Zheng W, Moritz KE, Ferrer-Alegre M, Cherry JJ, Androphy EJ, Wang A, Xu X, Austin C, Fischbeck KH, Marugan J, Burnett BG


JCI Insight , (1), e88427, 2016.

Article Pubmed
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Small molecule inhibitor of NRF2 selectively intervenes therapeutic resistance in KEAP1-deficient NSCLC tumors.


Singh A, Venkannagari S, Oh KH, Zhang Y, Liu L, Rohde J, Nimmagadda S, Sudini K, Brimacombe K, Gajghate S, Ma J, Wang A, Xu X, Shahane SA, Xia M, Woo J, A Mensah G, Wang Z, Ferrer-Alegre M, Gabrielson E, Li Z, Rastinejad F, Shen M, Boxer MB, Biswal S


ACS Chem. Biol. , 2016.

Article Pubmed

Identification of Multiple Cryptococcal Fungicidal Drug Targets by Combined Gene Dosing and Drug Affinity Responsive Target Stability Screening.


Park YD, Sun W, Salas A, Antia A, Carvajal C, Wang A, Xu X, Meng Z, Zhou M, Tawa G, Dehdashti J, Zheng W, Henderson CM, Zelazny AM, Williamson PR


MBio , (7), 2016.

Article Pubmed
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