Rare Disease Day - A Day to Raise Awareness & Drive Action🌟 In 2025, initiatives across multiple continents are bringing attention to rare diseases in diverse and impactful ways. 🖇️Check out what our member oganizations are preparing: irdirc.org/rare-disease-d…

🚀Wrapping up two inspiring days at CA-SC Meeting Over the past two days, experts, researchers, patient advocates, and industry leaders came together to tackle key challenges in RD research that have reinforced the power of collaboration. A huge thank you to all! 🤝

We are at RE(ACT) Congress & IRDiRC Conference 2025 in Brussels 🇧🇪 🌎Joined by experts from around the world, RE(ACT) offers a unique opportunity for research on rare diseases and orphan drugs. #Rarevolution #REACTCongress #IRDiRC #ERDERA #BLACKSWANFOUNDATION

Final day of the RE(ACT) Congress & @IRDiRC Conference! 💡🔬 Busy day ahead, looking at funding models and drug repurposing in #RareDiseases. Before the wrap-up, hear from participants about some of their key takeaways and insights. Watch now 🎥👇#RAREvolution

🚨 Call for Applications: Join the IRDiRC Therapies Scientific Committee! We're looking for 3 new members to help shape the future of rare disease treatment. If you have expertise in therapy development, apply by April 6, 2025! 📩 Apply: [email protected]

📑New IRDiRC Publication We are thrilled to announce that our latest article: "Drug–device combinations in rare diseases: Challenges and opportunities" is now available in Drug Discovery Today. 🔗 Read the full article: doi.org/10.1016/j.drud… #IRDiRC #RareDiseases #DDCs

🚨 Last chance to register! 🚨 Join Europe’s rare disease community in Warsaw on 10–11 April for the Towards an EU Action Plan on Rare Diseases conference! 💫 🔗 Register now 👉 loom.ly/ekq3FDg ⏳ Deadline: 3 April – Don't miss out!

📣 [NEW REPORT] A deep dive into the crucial role of industry in Public-Private Partnerships for #RareDiseases research! 🚀 📘 A must-read to understand industry’s impact in successful PPPs. Read the full report: irdirc.org/wp-content/upl… #PPP #RareDiseaseResearch #Innovation

📢 On April 25, EMA's CHMP gave a positive opinion for givinostat (Duvyzat) — a novel HDAC inhibitor for ambulant #DMD patients (6+ yrs) with corticosteroids. Final EC decision expected by July 2025. A key step for the #RareDisease community. #Givinostat #EMA

📣 New PACC Chair Announcement! 📣 We're thrilled to welcome Kelly du Plessis (RARE DISEASES INTERNATIONAL) as Chair of IRDiRC's Patient Advocacy Constituent Committee! 🔗 Learn more: irdirc.org/pacc #RareDisease #PatientAdvocacy #IRDiRC

⭐ New Funding Models Toolbox now live! ⭐ Helping #RareDisease advocates understand funder roles, goals & strategies. 🔧 Toolbox: lnkd.in/ezDB6b2q 📄 Companion paper: lnkd.in/eF9RiVPZ ℹ️ Task Force info: lnkd.in/ePYzPApp #RareDisease #Fundingstrategies

🚨 Major News from Geneva! 🚨 At #WHA78, WHO Member States adopted the 1st-ever Resolution on Rare Diseases — a huge win for 300M+ people worldwide. 🌍 🤝 Let’s keep moving — together. 📰Read the resolution: apps.who.int/gb/ebwha/pdf_f… #RareDiseases #HealthEquity #GlobalHealth

📢 Call for New Members: IRDiRC ISC! 🌍 We’re looking for experts in: 🔹 Ontologies/language learning 🔹 Pharmacy/pharmaceutical sciences 🔹 Health economics/HTA 🔹 Ethics 🗓️ Apply by 27 June 2025 📩 scientific.secretariat[at]irdirc.org 🔗 irdirc.org/isc/

⭐ New IRDiRC Publication: "From roadmap to a sustainable end-to-end individualized therapy pathway" ⭐ 🌍 Let’s make individualized therapies a standard, not an exception. 🔗 Read more: journals.sagepub.com/doi/10.1177/26… #RareDisease #Nof1 #PrecisionMedicine #IRDiRC

📢 Exciting news! Saumya Jamuar (@SingHealthDuke) has been elected Vice Chair of the IRDiRC Diagnostics Scientific Committee! 🧬👏 A leader in rare genetic disorders, Saumya brings deep expertise to the role. Welcome aboard! 🌍💡 🔗 irdirc.org/dsc/ #RareDisease #IRDiRC

⭐ IRDiRC at #WODC Europe 2025! ⭐ We're excited to join the largest global event for #orphandrugs & #rarediseases 🌍 📍 RAI Congress Centre, Amsterdam 🇳🇱 📅 Workshop: Oct 27 | Congress: Oct 28–29 Come visit our booth to learn more about #IRDiRC! More details to follow soon!

📣 Ongoing Call for New ISC Members! 👤 IRDiRC’s Interdisciplinary Scientific Committee is recruiting 3 new experts starting July 1, 2025. 🗓️ Apply by June 27, 2025 🧠 Commit to active participation in meetings & ISC work! 📬irdirc.org/irdirc-interdi… #RareDiseases #IRDiRC

🌟 New IRDiRC Newsletter published! 🌟 🧬 Read the latest news in rare disease research, policy & more. 📩 Read now: irdirc.org/wp-content/upl… 📲 Stay in the loop—subscribe here: irdirc.org/news-events/ir… #IRDiRC #newsletter #rarediseases

🚀 ERDERA launches its Networking Support Scheme! 🌍 🎯 Funding up to €30K/event for networking in rare diseases 🤝 Open to researchers, clinicians, advocates from 3+ ERDERA countries (1 underrepresented) 📅 Deadline: 7 Oct 2025 🔗 More details: erdera.org/nss/