Program

769
496
86
79
77
33
25
15
13
11
10
10
9
4
2

Journal Year Published

54
161
315
323
373
112
57
45
43
32
15
7
3
1
1
1
1
2
1
1

Author

159
147
113
109
105
104
103
100
94
72
67
67
58
50
49
35
34
34
30
29

Category

277
271
269
82
81
51
21
18
14
14
2
2

Keyword

1148
566
515
338
325
290
204
171
154
142
118
106
105
102
93
92
89
79
76
76

Journal

43
41
41
28
23
19
17
14
13
13
13
13
13
12
12
11
11
11
11
10

NCATS Publications

Synthesis and N-Methyl-d-aspartate (NMDA) Receptor Activity of Ketamine Metabolites.


Morris P, Moaddel R, Zanos P, Moore CE, Gould T, Zarate CA, Thomas C


Org. Lett. , (19), 4572-4575, 2017.

Article Pubmed
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Parallel Chemistry Approach to Identify Novel Nuclear Receptor Ligands Based on the GW0742 Scaffold.


Teske KA, Rai Bantukallu G, Nandhikonda P, Sidhu PS, Feleke B, Simeonov A, Yasgar A, Jadhav A, Maloney DJ, Arnold LA


ACS Comb Sci , (19), 646-656, 2017.

Article Pubmed
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Human Relaxin Receptor Is Fully Functional in Humanized Mice and Is Activated by Small Molecule Agonist ML290.


Kaftanovskaya EM, Soula M, Myhr C, Ho BA, Moore SN, Yoo C, Cervantes B, How J, Marugan J, Agoulnik IU, Agoulnik AI


J Endocr Soc , (1), 712-725, 2017.

Article Pubmed
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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.


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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Future of Rare Diseases Research 2017-2027: an IRDiRC Perspective.


Austin C, Cutillo C, Lau LP, Jonker AH, Rath A, Julkowska D, Thomson D, Terry S, de Montleau B, Ardigò D, Hivert V, Boycott KM, Baynam G, Kaufmann P, Taruscio D, Lochmüller H, Suematsu M, Incerti C, Draghia-Akli R, Norstedt I, Wang L, Dawkins HJ, International Rare Diseases Research Consortium (IRDiRC)


Clin Transl Sci , 2017.

Article Pubmed

Progress in rare diseases research 2010-2016: an IRDiRC perspective.


Dawkins HJ, Draghia-Akli R, Lasko P, Lau LP, Jonker AH, Cutillo C, Rath A, Boycott KM, Baynam G, Lochmüller H, Kaufmann P, Cam YL, Hivert V, Austin C, International Rare Diseases Research Consortium (IRDiRC)


Clin Transl Sci , 2017.

Article Pubmed
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Dual AAV gene therapy for Duchenne muscular dystrophy with a 7-kb mini-dystrophin gene in the canine model.


Kodippili K, Hakim C, Pan X, Yang HT, YUe Y, Zhang Y, Shin JH, Yang N, Duan D


Hum. Gene Ther. , 2017.

Article Pubmed

First Selective 12-LOX Inhibitor, ML355, Impairs Thrombus Formation and Vessel Occlusion In Vivo With Minimal Effects on Hemostasis.


Adili R, Tourdot BE, Mast K, Yeung J, Freedman JC, Green A, Luci D, Jadhav A, Simeonov A, Maloney DJ, Holman TR, Holinstat M


Arterioscler. Thromb. Vasc. Biol. , (37), 1828-1839, 2017.

Article Pubmed