Future of Rare Diseases Research 2017-2027: An IRDiRC Perspective

被引：162

作者：

Austin, Christopher P. ^{[1
]}

Cutillo, Christine M. ^{[1
]}

Lau, Lilian P. L. ^{[2
]}

Jonker, Anneliene H. ^{[2
]}

Rath, Ana ^{[2
,3
]}

Julkowska, Daria ^{[4
]}

Thomson, David ^{[5
]}

Terry, Sharon F. ^{[6
]}

de Montleau, Beatrice ^{[7
]}

Ardigo, Diego ^{[8
]}

Hivert, Virginie ^{[7
]}

Boycott, Kym M. ^{[9
]}

Baynam, Gareth ^{[10
,11
]}

Kaufmann, Petra ^{[1
]}

Taruscio, Domenica ^{[12
]}

Lochmuller, Hanns ^{[13
]}

Suematsu, Makoto ^{[14
]}

Incerti, Carlo ^{[15
]}

Draghia-Akli, Ruxandra ^{[16
,17
]}

Norstedt, Irene ^{[16
]}

Wang, Lu ^{[18
]}

Dawkins, Hugh J. S. ^{[19
]}

机构：

[1] NIH, NCATS, Bldg 10, Bethesda, MD 20892 USA

[2] INSERM, Rare Dis Platform, US14, IRDiRC Sci Secretariat, Paris, France

[3] INSERM, Rare Dis Platform, US14, Orphanet, Paris, France

[4] ANR, Paris, France

[5] Shire, Lexington, MA USA

[6] Genet Alliance, Washington, DC USA

[7] EURORDIS Rare Dis Europe, Paris, France

[8] Chiesi Farmaceut SpA, Corp Drug Dev, Parma, Italy

[9] Univ Ottawa, Childrens Hosp Eastern Ontario, Res Inst, Ottawa, ON, Canada

[10] King Edward Mem Hosp, Genet Serv Western Australia, Perth, WA, Australia

[11] Western Australian Register Dev Anomalies, Perth, WA, Australia

[12] Ist Super Sanita, Natl Ctr Rare Dis, Rome, Italy

[13] Newcastle Univ, Inst Med Genet, Newcastle Upon Tyne, Tyne & Wear, England

[14] Japan Agcy Med Res & Dev AMED, Tokyo, Japan

[15] Sanofi Genzyme, Cambridge, MA USA

[16] European Commiss, Directorate Gen Res & Innovat DG RTD, Brussels, Belgium

[17] Merck & Co Inc, Upper Gwynedd, PA USA

[18] NHGRI, NIH, Bethesda, MD 20892 USA

[19] Govt Western Australia, Off Populat Hlth Genom, Publ Hlth Div, Dept Hlth, Perth, WA, Australia

来源：

CTS-CLINICAL AND TRANSLATIONAL SCIENCE | 2018年 / 11卷 / 01期

关键词：

HEALTH DISPARITIES; ORPHAN DRUGS; PLATFORM;

D O I：

10.1111/cts.12500

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

100103 [病原生物学]; 100218 [急诊医学];

摘要：

[No abstract available]

引用

页码：21 / 27

页数：7

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