On October 14, 2023, the press conference of “Breaking the Last Mile – Research Report and Policy Recommendations on the Implementation of Rare Disease National Drugs (2023)” was successfully held […]
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The IQSEC2 Research and Advocacy Foundation will provide a 1-year grant, with a no cost extension of up to one year granted with approval, to support research related to IQSEC2-related […]
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The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal years (FY) 2023 – FY 2025 to support clinical trials for […]
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In the context of the Belgian Presidency of the Council of the European Union, the Belgian minister of health wants to make a focal point of identifying and using evidence […]
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The 2024 edition of the Open Academy Schools will take place in-person on 3-7 June in Barcelona. The face-to-face intensive week of the Open Academy School on Medicines Research & Development (formerly EURORDIS Summer School) […]
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Europe’s Most Exciting Orphan Drug Event The World Orphan Drug Congress is the largest and most established orphan drugs & rare diseases meeting globally. Now in its 14th annual edition, […]
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Following a positive opinion from the European Medicines Agency (EMA), the transfer of marketing authorization of Strimvelis from Orchard Therapeutics has been approved by the European Commission. Fondazione Telethon (Italy) […]
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Don’t miss out the new IRDiRC publication developed by Therapies Scientific Committee (TSC) members Daniel O’Connor, Annemieke Aartsma-Rus, Anneliene Jonker, and Regulatory Scientific Committee (RSC) member, Michela Galbado – “Defining […]
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New Publication: Making Rare Disease Research Attractive to Companies by IRDiRC Chrysalis Task Force
Accessible at https://doi.org/10.1177/26330040231188979, the Chrysalis Task Force identified key financial and non-financial factors that make rare disease research and development attractive to companies. More information on the Chrysalis Task Force […]
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Under the Horizon Europe Framework Programme, a new two-stage call for proposals for “Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases” was published on 27 July, […]
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