ERDERA

🚀 The countdown is on! This year, ERDERA will be present at the #WODC, the world’s leading event for #RareDiseases, bringing together 2,000+ attendees, 250+ speakers & 130+ exhibitors.
🗓️ 27–29 Oct 2025, Amsterdam
🔗More information at https://loom.ly/sG0eXT0

📢 Exciting news — the #ERDERA Networking Support Scheme is now open for applications! 👉 If you are working to advance research in #RareDiseases or rare cancers, this could be the funding opportunity you're looking for!
🔗 Find out more: https://loom.ly/CwIvcvM
🔁 Repost: Help us spread this info!

📢 Coming in May 2026: #EURORDIS Open Academy in Barcelona!
Applications open 8 Sept 2025 for the Medicines R&D and Scientific Innovation Schools.
🎓 80 fully funded spots via #ERDERA
🧪 Hands-on sessions
🤝 Network with advocates & researchers
🔗 https://loom.ly/oK6lwVI #RareDiseases

CRISPR-Cas: From biology to therapeutic applications A decade of groundbreaking research on bacterial CRISPR-Cas defence systems has culminated in the development of revolutionary editing technologies. This tool has been exploited to create transgenic …

🔬 CRISPR-Cas has transformed genome editing—from basic science to clinical therapies. Join experts at the EMBO Workshop to explore the latest advances in #CRISPR tech.
📍 Sorrento, Italy
📅 2–5 Nov 2025
🔗 https://loom.ly/G7HR4Eg

🚀 Want to lead the way in #RareDisease research?
Subscribe to #ERDERA’s newsletter! 📩
✅ Big news first
✅ Expert tips & tools
✅ Breakthroughs that matter
✅ Community highlights
Don’t miss a beat 👉 https://loom.ly/NxMGydc

Last chance to make your voice heard! 🗣️ #ERDERA has launched a survey to explore how #RareDisease patients can contribute to EU-funded research.
🔐 Confidential & 🤝 Co-created with patients
🔗 Take the survey: https://shorturl.at/ePVuL
📄 Learn more: https://shorturl.at/c03db

🚀 Registration is open for the FAIR Training Program 2025!
Learn how to make rare disease data more Findable, Accessible, Interoperable & Reusable. 🗓 24–26 Sept | 💻 Online | 🎓 Free
Limited places—register now 👉 https://loom.ly/8q3ieQQ
#FAIRdata #ERDERA #RareDiseases

📣 Do you know about #ERDERA's Networking Support Scheme? 👇
It funds events that boost rare disease & rare cancer knowledge exchange. 👩‍⚕️ For researchers, clinicians, advocates & more. 📅 Apply by 7 Oct 2025 🔗 https://loom.ly/CwIvcvM
#ResearchFunding #RareDiseases #RareCancers

🌍 Over 150 participants from 30 underrepresented countries joined the first #ERDERA Jamboree in Prague & online! The event spotlighted inclusion in #RareDisease diagnostic research & the need for equitable access to innovation. 🔗 Read more: https://loom.ly/GoLgpvk

A first in personalised medicine: CRISPR treatment for a baby sparks new debate on rare disease therapies - ERDERA In an unprecedented medical milestone, in February 2025, a six-month-old baby named K.J. Muldoon became the first person to receive a personalised CRISPR-based therapy tailored to correct a unique mutation causing carbamoyl phosphate synthetase 1 (CPS1) deficiency—a rare and deadly metabolic disorder. Developed, tested, and delivered in just six months, the therapy used base editing

👉 A first in personalised medicine: #CRISPR treatment for a baby sparks new debate on rare disease therapies. 🔗 Read the full article on this unprecedented medical milestone here: https://loom.ly/pYb-OuI #ERDERA

“ERDERA could become a model of how to align large-scale, cross-border research with real patient needs, so that the knowledge gained is translated into tangible benefits” - ERDERA My name is Tomasz Grybek. First—and foremost—I’m a father of a child living with a rare disease called metachromatic leukodystrophy. My son, Borys, is now a sixteen-year-old teenager, and we also have a younger daughter, Greta—she’s our sweetie and Borys’s little sister. The second “hat” I wear is my professional capacity: by training, I’m an

🎙️ “Research must align with real patient needs.” In our latest conversation, @TomaszGrybek —@EURORDIS board member and MetabERN advocate—shares how his family’s #RareDisease journey shaped his view on cross-border science.
🔗 Read more: https://loom.ly/BWwEPTg

Patients as Equal Partners – ERDERA’s PPIE Vision Echoes in Brussels - ERDERA Earlier this year, the International Patient Organisation for Primary Immunodeficiencies (IPOPI) hosted the forum “Shaping the Future of Research: Patients as Equal Partners”. Policymakers, researchers and advocates agreed that rare-disease programmes still lean too heavily on patient groups for feedback while denying them a real share of decision-making. Dr Daria Julkowska, Coordinator of the European

👥 #RareDisease #Patients as equal partners—in Brussels and beyond.
At @ipopi ’s Brussels forum, @erdera’s Dr Daria Julkowska set out a vision where lived experience shapes research from the start.🔗More: https://loom.ly/ifI80KE

“The notion that public-private collaboration is inherently problematic doesn’t align with current realities. Not only is it possible—it is necessary, beneficial, and more straightforward than many assume.” - ERDERA Earlier this year, you moderated a session at the RE(ACT) Congress on the many challenges in collaboration for rare diseases and hybrid funding models. What were your main takeaways from the discussion? I identified four key takeaways from the session. First, translating science into products is not a simple journey. No single funding source can

🤝 #PublicPrivate #collaboration isn’t a risk — it’s the route to faster #RareDisease therapies. In this interview, EFPIA’s Magda Cheblus explains how seamless EU funding paths and open dialogue can move discoveries from lab to life.
📄 Read on: https://loom.ly/3fUYfMY

📣 ERDERA is seeking specialists to join our expert pool! Get involved in activities, panels & networking, plus access funding, training & more.
Interested? 👉 https://loom.ly/YUHruUQ 🗣️ Share with those who might be interested!

Last chance to make your voice heard! 🗣️ #ERDERA has launched a survey to explore how #RareDisease patients can contribute to EU-funded research.
🔐 Confidential & 🤝 Co-created with patients
🔗 Take the survey: https://shorturl.at/ePVuL
📄 Learn more: https://shorturl.at/c03db

New Toolkit Strengthens Collaboration Between ERNs and Industry - ERDERA A new resource has been launched to help bridge the gap between European Reference Networks (ERNs) and life sciences companies, aiming to advance research and care for people living with rare diseases. The Together For Rare Diseases (T4RD) Toolkit, introduced at a joint webinar by Together4RD and ERDERA on 23 June 2025, offers practical guidance

📢 The new T4RD Toolkit by Together for Rare Diseases & #ERDERA is here! It supports collaboration between #ERNs and life sciences companies with practical guidance based on real rare disease community experience, including #MetabERN 🤝 🔗 Learn more: https://loom.ly/SeshMlU

At a recent high-level webinar hosted by Science Magazine and the Fondation Ipsen Daria Julkowska expressed #ERDERA's perspective to rare disease research ecosystem. 🔗 Read more about this insightful webinar: https://loom.ly/LRrEwx8

Thank you for sharing this important survey 🙌

Patient organisations: Make your voice heard! 🗣️ #ERDERA has launched a survey to explore how #RareDisease patients can contribute to EU-funded research.
🔐 Confidential & 🤝 Co-created with patients
🔗 Take the survey here: shorturl.at/ePVuL
📄 Learn more: shorturl.at/c03db @eurordis.bsky.social

Have your say: EU AI Act consultation on high-risk systems - ERDERA The European Commission has opened a public consultation to gather feedback on the implementation of the AI Act's rules concerning high-risk AI systems. The aim is to collect practical examples, clari...

🗣️ The #EU has opened a public consultation on how to implement #AI Act rules for high-risk systems. 🤝 Help shape future guidelines with your feedback. Read more here👉 loom.ly/6VB9xrQ #PublicConsultation #ERDERA @ec.europa.eu

New SOP to enhance transparency and quality of Real-World Data - ERDERA A new Standard Operating Procedure (SOP) titled “Validation, Publication, and Maintenance of Real-World Data Sources and Studies in the HMA-EMA RWD Catalogues” (SOP/TDA/3543) has been published by the...

📑 ‪the #EMA‬ has published a new Standard Operating Procedure (SOP) titled: “Validation, Publication, and Maintenance of Real-World Data Sources and Studies in the HMA-EMA RWD Catalogues” 📄 Learn more: loom.ly/rofu5E8 @ema.europa.eu

Triplet repeat expansion project sets benchmark for public-private collaboration - ERDERA One of the pilot projects launched under ERDERA’s predecessor, the European Joint Programme on Rare Diseases (EJPRD), has become a flagship example of successful public–private collaboration in rare d...

🧬 A pilot project launched under #ERDERA’s predecessor (#EJPRD) is now a reference model for public–private collaboration—accelerating drug discovery to detect #Genetic changes linked to 30+ rare neurological conditions. 🔗 Read more: loom.ly/Ro-hf6g

📣 Daria Julkowska, #ERDERA 's Scientific Coordinator, will be speaking at @science.org & Fondation Ipsen webinar: "Bridging Silos: How Scientists Studying #RareDisease Are Building Cross-Disease Communities to Advance Research and Innovation"
📅 25/06 at 6 p.m. CET
🔗 Register to join: loom.ly/1oH4heY

🔔 Join us on 23 June at 17:00 CEST for the launch of the #Together4RD Toolkit – a key resource to boost ERN-industry collaboration in #RareDisease research.
🔹 Practical tools
🔹 Real-world cases
🔹 Expert speakers
👉 Register: loom.ly/9TZyyX4
#ERDERA

📬 The latest IRDiRC newsletter is out!
Did you miss it? No worries — you can catch up on all the latest highlights, updates, and opportunities in the #RareDisease research community.
👉 Read it here: loom.ly/J3TUSKg
#ResearchCollaboration #IRDiRC #ERDERA

“There’s a lot of value in connecting with other countries — learning what has worked, what hasn’t, and applying those lessons” - ERDERA Gavin Lawler co-ordinates the Irish National Mirror Group (NMG) on rare diseases as Programme Manager within the Health Research Board Ireland. Established only in September 2024, this NMG brings toge...

💬 “There’s a lot of value in connecting with other countries.”
Ireland’s National Mirror Group unites policy, clinicians, researchers & patients to drive the new rare-disease strategy with #ERDERA.
🔗 Read the full interview: loom.ly/HSj6TtY

Make your voice heard! 🗣️ #ERDERA has launched a survey to explore how #RareDisease patients can contribute to EU-funded research.
🔐 Confidential & 🤝 Co-created with patients
🔗 Take the survey: shorturl.at/ePVuL (open until mid-July)
📄 Learn more: shorturl.at/c03db

“The National Mirror Group was the first time patients had been treated as equal partners in this kind of forum, and it immediately changed the conversation” - ERDERA Sandra Alves, a senior human-genetics researcher at the National Institute of Health Dr Ricardo Jorge (INSA), has coordinated Portugal’s National Mirror Group (NMG) for rare diseases since its creatio...

Interview 💬 | “It immediately changed the conversation” — Sandra Alves on giving #Patients equal footing in 🇵🇹's National Mirror Group.
Since 2021, Alves has helped align #Portugal's efforts with EU priorities through #ERDERA.
📖 Read the interview: loom.ly/qes6omw
#RareDiseases

📣 ERDERA is happy to announce a new facilitation period for the MOOC: “Diagnosing Rare Diseases: From the Clinic to Research and Back”!
📅 12 May – 4 July 👉 Get expert #ERDERA support, ask questions & boost your knowledge in #RareDiseases 🔗 More at loom.ly/88Of0Ps

Europe’s rare-disease alliance charts next steps at Riga workshop - ERDERA Delegates from 23 countries in Europe and beyond, together with scores of virtual participants, came together in Riga, Latvia, today for the European Rare Diseases Research Alliance (ERDERA) workshop ...

🌍 Delegates from 23 countries met in Riga for #ERDERA’s workshop to boost rare disease plans & research across Europe & beyond.
🤝 National Mirror Groups are key—linking ministries, researchers & patients. Target: 37 groups active by next year.
📲 More: loom.ly/CggG1mc