Program

130
29
10
7
7
3
2
2
1

Journal Year Published

2
9
9
7
7
12
13
17
17
12
33
21
8
3
3

Author

174
182
134
134
125
118
116
114
108
88
86
81
73
56
51
50
43
43
42
34

Category

78
66
29
29
23
11
8
8
6
4
1

Keyword

99
49
36
35
32
24
23
20
17
16
14
14
13
12
12
11
11
10
10
10

Journal

12
10
9
8
8
7
7
5
5
4
4
4
4
3
3
3
3
3
2
2

NCATS Publications

Application of a Dynamic Map for Learning, Communicating, Navigating, and Improving Therapeutic Development.


Wagner JA, Dahlem AM, Hudson LD, Terry SF, Altman RB, Gilliland CT, DeFeo C, Austin C


Clin Transl Sci , 2017.

Article Pubmed

Sample image from publication page

A dynamic map for learning, communicating, navigating and improving therapeutic development.


Wagner J, Dahlem AM, Hudson LD, Terry SF, Altman RB, Gilliland CT, DeFeo C, Austin C


Nat Rev Drug Discov , 2017.

Article Pubmed

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The International Rare Diseases Research Consortium: Policies and Guidelines to maximize impact.


Lochmüller H, Torrent I Farnell J, Le Cam Y, Jonker AH, Lau LP, Baynam G, Kaufmann P, Dawkins HJ, Lasko P, Austin C, Boycott KM, IRDiRC Consortium Assembly


Eur. J. Hum. Genet. , 2017.

Article Pubmed

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Voluntary Site Accreditation - Improving the Execution of Multicenter Clinical Trials.


Johnston SC, Austin C, Lewis-Hall F


N. Engl. J. Med. , (377), 1414-1415, 2017.

Article Pubmed

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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The importance of international collaboration for rare diseases research-a European perspective.


Julkowska D, Austin C, Cutillo C, Gancberg D, Hager C, Halftermeyer J, Jonker AH, Lau LPL, Norstedt I, Rath A, Schuster R, Simelyte E, van Weely S


Gene Ther. , 2017.

Article Pubmed