IRDiRC

🇪🇺 @eurordis launches Europe’s Blueprint for Rare Diseases, calling for a coordinated EU Action Plan to improve equitable access to care and expertise across Europe. 🔗eurordis.org/publications/eu… #RareDiseases #EURORDIS #RDBlueprint

📚 Why do some rare diseases remain overlooked? A new study from the IRDiRC PLUTO mission reveals major gaps in research and drug development: 🔹 50% had publications but no clinical development 🔹 Median wait for orphan drug designation: 45 years 👉 link.springer.com/article/10…

🚨 Applications open! Join the RDI Youth Leadership Programme 2027–2028 to grow your leadership, connect with young rare disease advocates worldwide and drive change! 🌍 Apply by 22 Nov 2026: form.typeform.com/to/wYYV0gG… #RDIYLP #RareDiseases

IRDiRC was delighted to see its work on #Nof1 treatments highlighted by Annemieke Aartsma-Rus (LUMC) at the #ERDERA 3rd General Assembly Meeting in Coimbra🇵🇹. 🔗 irdirc.org/preparing-for-gen…

🌟 Build relationships that move your mission forward! @GlobalGenes & Termeer Institute launch Leadership Lab to empower rare disease advocacy leaders. 💡Learn more: globalgenes.org/rare-leaders… #RareDisease #PatientAdvocacy

📣 New publication: Precompetitive collaboration can accelerate rare disease R&D by enabling data sharing, co-creating standards and tools, reducing duplication, and speeding innovation. Discover key success factors and real-world examples. 🔗 sciencedirect.com/science/ar…

🌍 IRDiRC welcomes Grace Andrews 🇱🇷 and Mohammed Harris 🇺🇸 to its Diagnostics Scientific Committee! Together, they’ll help advance rare disease diagnosis, genomics, data sharing and shorten the diagnostic odyssey. #IRDiRC #RareDisease

📣 New publication! How can young people with rare diseases become meaningful research partners? An IRDiRC perspective offers practical guidance for developmentally appropriate, ethical engagement. 🔗frontiersin.org/journals/ped…

🌍 IRDiRC was present at Sanofi’s Rare Dialogues in Paris, bringing regional voices together to advance rare disease research and care. 🤝 Collaboration and patient voices are key to global progress. #RareDiseases #IRDiRC #RareDialogues

📣 RDI Webinar: Innovative Financing for Rare Diseases. Join the final webinar session, as experts explore how financing solutions can move from concept to implementation. 🗓️ 29 Sept | 14:00 CEST 👉events.teams.microsoft.com/e…

📢 Final reminder! Poster Zone applications for #WODCEurope 2026 close Monday, 28 Sept. Showcase your research to the global rare disease community. 🧬 Apply: terrapinn.com/conference/wor… 📍 Amsterdam | 26–28 Oct

🚀 Registrations are open for #REACTCongress2027! Join the rare disease community in Budapest, 10–12 March 2027, for 3 days of science, collaboration & innovation. 🔗 react-congress.org/ #RareDiseases #RAREvolution #REACTcongress

🌟 Rare disease patient group leaders: make your voice heard! Share your experience in @RareBeacon Rare Insights Study and help shape evidence that can support funding, impact and service delivery across the UK. Take the survey by 19 October: 👉 rarebeacon.org/rare-insights…

🌟 Welcome to IRDiRC’s new Interdisciplinary Scientific Committee members! Elizabeth Hart 🇺🇸 (FDA) and Teguh Haryo Sasongko 🇲🇾 (IMU University) bring valuable expertise and global perspectives to rare disease research. 🤝 💡Learn more: irdirc.org/isc/ #IRDiRC

🔎 Rare Disease Day 2027 launches soon! Join EURORDIS’ campaign webinars on 19 Oct (4pm CEST) or 20 Oct (9am CEST) to discover the concept, heroes & resources. 🌍 💡More information: eurordis.org/rare-disease-da… #RareDiseaseDay2027

🎙️The SCGE Workshop will take place on 29 Sept 2026, exploring clinical delivery, regulation & payment models for individualized genome editing therapies. Join the discussion on enabling sustainable patient access. 📩 Register now: scge.mcw.edu/scge-workshop/

🌐 New event: EMA & HMA workshop on real-world data in Duchenne muscular dystrophy, 25 Sept 2026. Shekhar Natarajan, Chair of IRDiRC’s Therapies Scientific Committee, will join stakeholders in person. #DMD #RWD #RareDiseases

🌟 PETAL Consortium brings together 100+ investigators across 20+ international sites to advance research in peripheral T-cell lymphoma (PTCL), fostering collaboration, data sharing, and discovery to better understand PTCL. 🔬🌎 💡Learn more: petalconsortium.org/

🌏 Expanding Rare Disease Care Across the Pilbara Six months ago, the Rare Care Centre in Western Australia commenced its Pilbara Hub clinics, bringing cross-sector rare disease care coordination to communities up to 1,500 kilometres from Perth, Australia.📍

⭐ IRDiRC at World Orphan Drug Congress Europe 2026! Join 1,800+ rare disease leaders in Amsterdam, 27–28 Oct. 🎟️ Early Bird: save 20% by 4 Sept: terrapinn.com/conference/wor… 📣 Poster Zone applications are open! #IRDiRC #WODC2026