دورية أكاديمية
The importance of international collaboration for rare diseases research: a European perspective.
| العنوان: | The importance of international collaboration for rare diseases research: a European perspective. |
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| المؤلفون: | Julkowska D; Department of Health &Biology, Agence Nationale de la Recherche, Paris, France., Austin CP; National Center for Advancing Translational Sciences (NCATS), US National Institutes of Health (NIH), Bethesda, MD, USA., Cutillo CM; National Center for Advancing Translational Sciences (NCATS), US National Institutes of Health (NIH), Bethesda, MD, USA., Gancberg D; Directorate Health, Directorate-General for Research and Innovation, European Commission, Brussels, Belgium., Hager C; Directorate Health Systems, Medical products and innovation, Directorate-General for Health and Food Safety, European Commission, Brussels, Belgium., Halftermeyer J; Department of Health &Biology, Agence Nationale de la Recherche, Paris, France., Jonker AH; IRDiRC Scientific Secretariat, Inserm US14, Paris, France., Lau LPL; IRDiRC Scientific Secretariat, Inserm US14, Paris, France., Norstedt I; Directorate Health, Directorate-General for Research and Innovation, European Commission, Brussels, Belgium., Rath A; Inserm US14-Orphanet, Paris, France., Schuster R; DLR Project Management Agency, Health, Bonn, Germany., Simelyte E; Directorate Health, Directorate-General for Research and Innovation, European Commission, Brussels, Belgium., van Weely S; The Netherlands Organisation for Health Research and Development (ZonMw), The Hague, The Netherlands. |
| المصدر: | Gene therapy [Gene Ther] 2017 Sep; Vol. 24 (9), pp. 562-571. Date of Electronic Publication: 2017 Jul 27. |
| نوع المنشور: | Journal Article; Review; Research Support, Non-U.S. Gov't |
| اللغة: | English |
| بيانات الدورية: | Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: London : Nature Publishing Group Original Publication: Houndmills, Basingstoke, Hampshire, UK : Macmillan Press Ltd., c1994- |
| مواضيع طبية MeSH: | International Cooperation*, Biomedical Research/*organization & administration , Rare Diseases/*therapy, Biomedical Research/economics ; European Union ; Humans ; Rare Diseases/diagnosis |
| مستخلص: | Over the last two decades, important contributions were made at national, European and international levels to foster collaboration into rare diseases research. The European Union (EU) has put much effort into funding rare diseases research, encouraging national funding organizations to collaborate together in the E-Rare program, setting up European Reference Networks for rare diseases and complex conditions, and initiating the International Rare Diseases Research Consortium (IRDiRC) together with the National Institutes of Health in the USA. Co-ordination of the activities of funding agencies, academic researchers, companies, regulatory bodies, and patient advocacy organizations and partnerships with, for example, the European Research Infrastructures maximizes the collective impact of global investments in rare diseases research. This contributes to accelerating progress, for example, in faster diagnosis through enhanced discovery of causative genes, better understanding of natural history of rare diseases through creation of common registries and databases and boosting of innovative therapeutic approaches. Several examples of funded pre-clinical and clinical gene therapy projects show that integration of multinational and multidisciplinary expertize generates new knowledge and can result in multicentre gene therapy trials. International collaboration in rare diseases research is key to improve the life of people living with a rare disease. |
| References: | Hum Mutat. 2015 Oct;36(10 ):915-21. (PMID: 26295439) Eur J Hum Genet. 2017 Feb;25(2):162-165. (PMID: 27782107) Nucleic Acid Ther. 2017 Feb;27(1):1-3. (PMID: 27929755) |
| تواريخ الأحداث: | Date Created: 20170426 Date Completed: 20180207 Latest Revision: 20220331 |
| رمز التحديث: | 20240628 |
| مُعرف محوري في PubMed: | PMC5628265 |
| DOI: | 10.1038/gt.2017.29 |
| PMID: | 28440796 |
| قاعدة البيانات: | MEDLINE |
Find this article in full text from Springer Verlag. 
Full Text Finder | تدمد: | 1476-5462 |
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| DOI: | 10.1038/gt.2017.29 |