for Executive Committee, Scientific Committees, and Working Groups

Upcoming IRDiRC meetings and teleconferences

• March 16, 2015: Executive Committee meeting in Madrid, Spain

• June 5, 2015: Diagnostics Scientific Committee meeting in Glasgow, UK (full day)

• June 5, 2015: Interdisciplinary Scientific Committee meeting in Glasgow, UK (full day)

• June 5, 2015: Therapies Scientific Committee meeting in Glasgow, UK (afternoon)

• June 6, 2015: Three Scientific Committees joint meeting in Glasgow, UK (morning)

• September 11, 2015: Executive Committee meeting in Montreal, Canada


Research highlights published on the website

 EMA chooses six medicines in the first phase of the adaptive pathways pilot project

 IRDiRC label to highlight research projects funded by an IRDiRC member

 Clinical trial designs for rare diseases: International Rare Cancers Initiative portfolio

 Drug repurposing based on a new concept: Homopharma

 Eleven centers designated as Genomic Medicine Centers to decode 100,000 genomes in the UK

 Telethon 2014 collects a record € 82,353,996 in France

 OPEN Act: legislation introduced in US congress to encourage research on drug repurposing for rare diseases

 Rethinking drug development for rare diseases: a combinatorial approach to treat Charcot-Marie Tooth disease

 The Canadian Rare Diseases Models and Mechanisms network has been awarded CAD 2.3 million by the Canadian Institutes of Health Research

 International Charter of principles for sharing bio-specimens and data

 ClinRegs: an online database of clinical research regulatory information

 Clinic-developed software to support NIH/NCATS global rare diseases registry

 HHS and NIH take steps to modify clinical trial rules to improve transparency, following initiatives from the WHO and EMA

 The 204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy


IRDiRC-related calls

• The ‘JPco-fuND‘ Joint Transnational call for European research projects on neurodegenerative diseases (risk and protective factors, longitudinal cohort approaches and advanced experimental models) was launched on 8 January 2015, in partnership with the European Commission under the ERA-NET Co-fund scheme, and will be open for pre-proposals until 10 March 2015.


Website updates

• The IRDiRC in the news section has been updated with the addition of three articles mentioning IRDiRC from Life Sciences ConnectFederal Telemedicine News and the International Society for Neonatal Screening.



Antonia Mills, IRDiRC Scientific Secretariat, IRDiRC, Plateforme Maladies Rares / Rare Disease Platform, 96 rue Didot, 75014 PARIS, FRANCE,           Tel: +33 1 56 53 81 37, Fax: +33 1 56 53 81 38