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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 6h

💬 “The report of the Rare Barometer survey… provided me with validation and a new frame of reference.” — Marliena, patient and advocate

Now it’s your turn.
✍️ Take part: tiny.cc/RB-MH

Child laughing and playing with bubbles outdoors beside text reading ‘What helps you live well? Take the survey.’ Logos for Rare Barometer and EURORDIS Rare Diseases Europe.

EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 3d

🧬 The #EUBiotechAct represents a key opportunity to address barriers and ensure that innovation translates into both improved patient outcomes and industrial resilience.

🗓️ Have your say in the public consultation before 10 November.

🔗 Read more: https://go.eurordis.org/BiotechAct

Close-up of a pipette releasing a blue liquid drop into a clear well in a laboratory test tray.

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 3d

💫 A wonderful close to the #ERDERA General Assembly!

Session 8: Training highlighted how the #EURORDISOpenAcademy empowers patient advocates & drives rare disease research across Europe by fostering learning, collaboration & impact. 💙

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 4d

Are you 18–30 and living with (or supporting someone with) a #RareDisease?

💬 We’re looking for a European youth advocate to join the #RareDiseaseDay 2026 Raising Youth Voices project!

👉 Learn more and apply now: https://go.eurordis.org/YouthVoices

Deadline to apply: 9 November

#YouthAdvocacy

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 4d

🎉 It’s been just over a year since the launch of #ERDERA, the European Rare Diseases Research Alliance!

At the #ERDERA General Assembly, Roseline Favresse (EURORDIS) spoke on patient & public involvement — ensuring research truly reflects patient needs. 💙
👉 https://erdera.org/

Panel of six speakers presenting on Patient and Public Involvement and Engagement in a university lecture hall with audience members using laptops.

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 5d

Across the three days, EURORDIS experts showcased how policy innovation, data-driven insights, and meaningful patient engagement. (8/8) #WODC #WODC2025

👉 Learn more about what went on at the ‌World Orphan Drug Congress Europe 2025: go.eurordis.org/k1YvAD

#wodc #wodc2025 #rarebarometer #wodc2025 #rarediseases #eurordis #patientadvocacy #healthpolicy #orphandrugs #lifesciences | EURORDIS-Rare Diseases Europe

At this year’s World Orphan Drug Congress Europe (WODC) 2025, EURORDIS colleagues shared their expertise across key sessions on policy, research, and patient advocacy. #WODC #WODC2025 During the pre-...

go.eurordis.org

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 5d

🧠 Finally, Matt Bolz-Johnson spoke on the importance embedding psychological support in R&D. He highlighted how designing clinical research and therapeutic pathways that integrate psychological support can significantly improve the health outcomes of those with rare diseases. (7/8)

Matt pictured smiling in a selfie with his fellow panellists.

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 5d

🏥 Roseline Favresse also shared findings from the #RareBarometer survey on diagnosis. She noted that access to diagnosis is faster when patients are referred to centres of expertise, underscoring the importance of connecting them through national and European Reference Networks. (6/8)

5 people sit on stools and laugh as Roseline speaks into a microphone.

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 5d

🤝 Jessie Dubief then presented our #RareBarometer programme, explaining that these studies enable the creation of evidence-based, patient-centred policies. She demonstrated how industry can partner with patient organisations to help them maximise the impact of their own data. (5/8)

Jessie speaks behind a lectern at the World Orphan Drug Congress.

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 5d

💬 Later that day, Julien Delaye contributed to a session on navigating the new Joint Clinical Assessments (JCAs), underlining the importance of patients’ expertise in JCAs and highlighting that patients hold essential real-world data that can strengthen these assessments. (4/8)

4 panellists sit in chairs in a wood-panelled room. Julien Delaye speaks into a microphone as the others watch.

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 5d

💡 On Tuesday, Rosa Catro joined a keynote panel exploring the future of Europe’s Life Science Strategy, stressing the urgent need for an EU Action Plan for Rare Diseases and emphasising the opportunities prevented by the EU Life Science Strategy and the upcoming Biotech Act. (3/8)

6 panellists and a moderator sit in chairs on stage looking at Rosa as she speaks into a microphone.

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 5d

During the pre-congress workshops, Rita Francisco, spoke on accelerating rare diseases drug development. (2/8)

🗣️“If we involve all stakeholders from the start, it is an implementation booster. All IHI projects have a responsibility to show success, and success stories persuade.”

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 5d

At this year’s World Orphan Drug Congress Europe (#WODC) 2025, EURORDIS colleagues shared their expertise across key sessions on policy, research, and patient advocacy. #WODC2025 #RareDiseases

Here are the key takeaways 🧵⬇️ (1/8)

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 6d

At the #ERNRND Annual Meeting, Ines Hernando, EURORDIS ERN & Healthcare Director, presented the European Rare Disease Lighthouses model, advancing access to specialised care for people with rare/complex conditions in Europe 🌍

A speaker stands at a podium presenting to a group seated in a modern, circular conference room with wood-paneled walls and large windows letting in natural light. The audience members are listening attentively, some taking notes, while the presenter speaks near a microphone and computer setup.

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 6d

🎉 20 years of dialogue. 40 workshops of impact.

Last week, at its 40th edition, the #EURORDIS Round Table of Companies spotlit Patient Experience Data as the key to patient-centred innovation.

Read more: go.eurordis.org/EurordisDial...

A diverse group of people seated in a circle during a workshop or discussion session in a wooden-panelled room with natural light. One participant uses a wheelchair, and a woman standing on the right is speaking to the group.

Audience seated in a bright conference room watching a presentation projected on a large screen. A man in the foreground is taking a photo of the slide with his phone.

Conference attendees facing a stage where speakers are seated in front of a large screen displaying the title “Reporting from Brainstorming Sessions.” The room has high wooden walls and a skylight ceiling.

People seated around round tables in a warmly lit restaurant with elegant chandeliers and greenery overhead. A woman stands and speaks into a microphone while others listen and smile.

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EURORDIS-Rare Diseases Europe @eurordis.bsky.social · 7d

Every #ECRD poster tells a story of research, collaboration & action for people living with a rare disease.

Share yours at #ECRD2026 and help shape the future of rare disease care in Europe.

📅 Deadline: 6 March 2026
👉 Submit: https://go.eurordis.org/ECRD2026Posters

"Submit a poster abstract!" A woman in a beige blazer and backpack attentively reads scientific posters displayed at a conference venue with large windows.

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