Program

127
27
10
7
7
3
2
2
1

Journal Year Published

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

Author

163
65
56
50
49
46
34
31
25
23
22
19
16
15
14
14
13
13
13
9

Category

74
60
28
24
23
10
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
7
7
7
4
4
4
3
3
3
3
3
3
3
2
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
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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

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
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