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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We are delighted to announce that IRDiRC Therapies Scientific Committee (TSC) Vice Chair, Anneliene Jonker (University of Twente, The Netherlands), will give a talk on the drug repurposing guidebook at […]
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Taking place in February to mark the occasion of Rare Disease Day, the EURORDIS Black Pearl Awards celebrate the inspirational qualities of people living with a rare disease along with […]
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FDA’s Rare Disease Endpoint Advancement (RDEA) Pilot Meeting Program is accepting RDEA proposals for this quarter until September 30, 2023. The RDEA Pilot Meeting Program is designed to support novel […]
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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 […]
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