EURORDIS-Rare Diseases Europe

EURORDIS-Rare Diseases Europe An alliance of patient organisations improving the lives of all people living with rare diseases.

🧬 Multi-omics is often described as one of the most promising frontiers in healthcare and research. But what could it me...
11/06/2026

🧬 Multi-omics is often described as one of the most promising frontiers in healthcare and research. But what could it mean for people living with rare diseases?

In her latest staff blog, our colleague Claudia Fuchs, EURORDIS Senior Project Manager on Therapeutic Development, explores how multi-omics could transform the way rare diseases are understood, diagnosed and treated by bringing together different layers of biological and environmental data to create a more complete picture of health and disease.

But scientific potential alone is not enough.

From data sharing and global collaboration to diversity, equity and patient involvement, Claudia examines the key challenges that must be addressed to ensure these advances benefit all people living with a rare disease, not just a few.

🔗 Read the blog and discover why the future of rare disease research depends not only on innovation, but also on inclusion: https://go.eurordis.org/staffblog

10/06/2026

⏰ Only 8 days left to register!

What does the latest survey tell us about the mental health and wellbeing of people living with rare or undiagnosed conditions?

On 18 June, we will unveil findings from more than 10,700 respondents worldwide and discuss the realities behind the data, from isolation and uncertainty to gaps in mental health and social support.

These results tell an urgent story. Join us to hear what they reveal and what must change to better support people living with rare conditions and their families.

🗓️ 18 June 2026
🕒 15:00–17:00 CEST
📍 Open to all

👉 Register here: https://go.eurordis.org/RBresults

08/06/2026

Last year, we celebrated some of the most inspiring changemakers in the rare disease community. Now it's your turn to shine a spotlight on the people making a difference!

🏆 Nominations for the 2027 EURORDIS are officially open!

From patient advocates and researchers to policymakers, organisations and people living with a rare disease, the Black Pearl Awards honour those whose dedication, innovation and commitment are helping transform lives across the rare disease community.

Do you know someone who deserves recognition?
Has a person, organisation or initiative inspired you?
Could that person be you?

Don't miss your chance to nominate your star of the rare disease community, or put yourself forward!

🔗 Submit your nomination today: https://go.eurordis.org/BPA2027

The rare disease community grows stronger when knowledge, experience, and lived realities are shared.There is still time...
06/06/2026

The rare disease community grows stronger when knowledge, experience, and lived realities are shared.

There is still time to apply as a mentor for the Open Academy x ERDERA Mentoring Programme - an opportunity to support patient groups and emerging leaders across the rare disease ecosystem.

Mentoring is not about having all the answers. It is about sharing lessons learned, encouraging independent thinking, and helping build a more connected and empowered community. Whether your experience comes from advocacy, patient organisation leadership, healthcare or research, your insights could help others navigate challenges and move forward with greater confidence!

🌍 Flexible online format
🗓️ December 2026 – May 2027
⏱️ Approximately 1 hour per month

⏳ Applications remain open for a limited time.

🔗 Apply now: https://go.eurordis.org/mentorcall

04/06/2026

Two days. Hundreds of voices. One verdict.

As closes in Prague, one thing is clear: across patients, policymakers, researchers, healthcare professionals and industry, there is now a rare and genuine consensus on what Europe must do for the 30 million people living with a rare disease, and on the fact that Europe is capable of doing it.

The discussions surfaced both hard truths and real momentum: fragmentation, unequal access to diagnosis, care and treatment, and the urgent need for stronger data, sustainable investment and meaningful patient involvement at every level of decision-making.

EURORDIS President Avril Daly was unequivocal: "Rare diseases are not niche." The community, she promised, would not stop "until every person living with a rare disease in Europe can access the diagnosis, care and treatment they deserve."

From the European Blueprint for Rare Diseases to policymakers from right across EU institutions and national governments pushing for binding legislation, the momentum from Prague is undeniable. The challenge now is not defining what needs to be done, but guaranteeing that Europe delivers it. ✊

📖 Read our conference wrap-up: https://lnkd.in/e2easM-N

04/06/2026

Day 2 of is underway!

This morning's parallel sessions brought together patients, clinicians, researchers and policymakers to identify priorities for strengthening rare disease care across Europe. Here are some of the key outcomes.

🏥 Access to Specialised Healthcare
Participants called for stronger coordination between local, national and European systems, clearer pathways to specialised care, and closer integration between national networks and European Reference Networks. Reducing the fragmentation that too often leaves patients and families navigating complex systems alone emerged as a shared priority.

💊 Access to Innovation
As Europe prepares for the extension of the HTA Regulation to orphan medicines in 2028, participants reinforced the need for patient involvement to become meaningful and systematic in decision-making. Recognising lived experience alongside clinical evidence, and ensuring innovation is not overlooked when data are limited, were identified as essential conditions for frameworks that work for rare diseases.

💚 Mental Health and Wellbeing
A clear call emerged to move beyond fragmented, episodic support and towards lifelong, integrated approaches that treat mental health as an essential component of rare disease care, not an afterthought. Prevention, resilience and quality of life must sit alongside physical health throughout the care journey.

Across all three sessions, a common conclusion took shape: improving outcomes for people living with rare diseases requires healthcare systems that are more coordinated, more person-centred, and better equipped to respond to the realities of rare disease throughout life.

👉 Want to be part of the discussion? The conference isn't over yet. Join us online and follow the remaining sessions as the rare disease community continues to shape the future of policy, care and innovation in Europe: https://go.eurordis.org/register-in

🤝 New connections, fresh ideas, and meaningful conversations.Beyond today’s sessions, both in Prague and online, the d...
03/06/2026

🤝 New connections, fresh ideas, and meaningful conversations.

Beyond today’s sessions, both in Prague and online, the day was marked by the exchange of ideas, experiences, and expertise. Through poster pitches, discussion tables, networking activities, and countless informal conversations, participants connected across disciplines, sectors, national borders, and disease areas to share knowledge and build new collaborations.
These exchanges are at the heart of . They remind us that progress in rare diseases is driven not only by scientific and policy advances, but also by the strength of a community working together towards a common goal.

As we head into Day 2, we're looking forward to continuing the conversations that will help shape a competitive future for rare disease policy, research, and care in Europe. 👋
👉 Get involved: https://go.eurordis.org/register-in

Three tracks. One shared message: patient voices, robust evidence, and cross-sector collaboration must be at the centre ...
03/06/2026

Three tracks. One shared message: patient voices, robust evidence, and cross-sector collaboration must be at the centre of Europe's rare disease agenda.

At , discussions across our first three parallel sessions highlighted key priorities for the future of rare diseases in Europe:

🔬 Shaping the Future of Rare Disease Therapies
Speakers stressed the need to build on existing assets: from ERNs and ERDERA to public-private collaborations and patient-led innovation. Stronger coordination across research, regulation, HTA and access pathways, meaningful patient involvement, and investment in education and workforce skills will be essential to turn scientific advances into therapies that reach patients.

👶 Early Detection & Newborn Screening
Expanding newborn screening and improving access to early genetic diagnosis can shorten the diagnostic odyssey and open pathways to timely care. Panellists highlighted the growing feasibility of genomic screening, the importance of European collaboration and harmonisation, and the need to ensure that people living with undiagnosed conditions remain visible, supported and included in research.

🫂 Advancing Holistic Care for Rare Conditions
Delivering better outcomes means looking beyond clinical care alone. Discussions focused on the value of patient-reported evidence, experience measures, registries and real-world data to better understand the full impact of rare diseases.

Interested in the topics discussed here? You can still register to join ECRD online and take part in the second session of these conference tracks, taking place later this afternoon.
👉 Get in on the action: https://go.eurordis.org/register-in

02/06/2026

What comes next for rare disease policy in Europe and globally?

Today, ahead of in Prague, the rare disease community is taking an important step towards answering that question through a dedicated workshop on the current draft of the European Blueprint for Rare Diseases.

Developed through a multi-stakeholder process led by EURORDIS and Rare Diseases International, the Blueprint brings together the actions and measures needed to address persistent inequities and improve the lives of more than 30 million Europeans living with rare diseases.

Today’s workshop is an opportunity to present the Blueprint so far, gather feedback from patients, advocates, healthcare professionals, researchers and policymakers, address outstanding issues, and identify priority actions for meaningful progress.

🚩 The final European Blueprint for Rare Diseases will be published by September and presented to key policymakers, informing both a future EU Action Plan on Rare Diseases and contributing to international efforts towards a WHO Global Action Plan.

📌 Over the coming days, we’ll be sharing more from as the rare disease community comes together to shape the future of rare disease policy, care and research. Join us online: https://go.eurordis.org/ECRDprogramme

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