Program

769
533
98
85
83
35
26
15
15
11
10
10
9
4
2

Journal Year Published

28
88
169
317
325
375
117
61
47
47
34
17
8
4
4
3
2
2
2
2

Author

165
164
120
117
114
110
105
105
104
77
72
71
61
52
50
41
39
37
33
31

Category

314
306
283
95
85
67
21
18
15
14
3
2

Keyword

1185
579
526
359
331
294
206
180
158
143
121
113
108
105
96
94
90
80
79
76

Journal

46
45
41
28
25
21
18
17
15
14
14
13
13
13
13
13
12
12
11
11

NCATS Publications

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

Sample image from publication page

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

Drug discovery and development for rare genetic disorders.


Sun W, Zheng W, Simeonov A


Am. J. Med. Genet. A , (173), 2307-2322, 2017.

Article Pubmed

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