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From 25–28 May 2026, Barcelona will host the next EURORDIS Open Academy Schools. Applications for the Medicines Research & Development School and the Scientific Innovation & Translation School open on 8 September 2025. Subscribe to the Open Academy and EURORDIS newsletters to receive the link as soon as the call goes live. From 25–28 May 2026, Barcelona will host the next EURORDIS Open Academy Schools. Applications for the Medicines Research & Development School and the Scientific Innovation & Translation School open on 8 September 2025. Subscribe to the Open Academy and EURORDIS newsletters to receive the link as soon as READ MORE

The FAIR Training Program is now open for registration. The online event is jointly organised by ERDERA and their associated partners and hosted by the World Duchenne Organization. The FAIR Training Program 2025 – 2027 falls under the framework of ERDERA and aims to equip participants with theoretical understanding and practical tools for implementing the FAIR data principles – Findability, Accessibility, Interoperability, and Reusability – in the context of health research and rare diseases. This training event is part of a series of three annual training opportunities to master FAIR principles in practice. The first event will take place online READ MORE

ERDERA, the European Rare Disease Research Alliance, has launched the Exploring RD Researchers’ Needs for Consultancy Services survey to map in which areas Rare Disease (RD) research community across Europe and beyond need external consultancy support most. Its findings could help shape a new dedicated ERDERA consultancy service offering specialised advice to projects funded through the partnership and the broader RD research community.  The survey, which is now open and accessible here, asks research community to pinpoint the gaps they encounter when seeking external expert consultancy support. Whether the support was requested in ethics and regulatory science areas, data readiness, READ MORE

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 Rare Diseases Research Alliance (ERDERA), backed that call. During the event, she proposed working with umbrella groups and ultra-rare associations to refine eligibility criteria so that dissemination, data stewardship and lived experience count alongside laboratory science. Why PPIE Matters for Families Living with a Rare READ MORE

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 economist, and I work as a quality and food safety manager in the food sector. The third capacity I have is patient advocacy, which I took on thanks to—or because of—Borys and his journey. I serve as a patient representative to ERN-ITHACA and MetabERN, and READ MORE

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 support the entire path from discovery to delivery. Therefore, there is a need for multiple funding tools: public, private, and hybrid. Second, these funding tools are currently fragmented, leading to a lack of continuity across research phases. Researchers often face disruptions when a grant ends, READ MORE

The EURORDIS Open Academy is set to host its 2025 Schools from 2–5 June in Barcelona, bringing together a diverse cohort of patient advocates and young researchers from across Europe.

ERDERA’s patient-engagement working group has opened an online survey to gauge how rare-disease patient organisations experience their role in publicly funded research, including projects financed under ERDERA’s predecessors, EJPRD and the E-Rare schemes. The questionnaire—already available here—will stay live for six to eight weeks and seeks first-hand assessments of what works, and what falls short, when patients are invited to shape studies that ultimately affect their own lives. Patients, care providers and advocates in the rare-disease field often acquire in-depth knowledge of their conditions, combining technical insight with lived experience. “Their perspectives are recognised as vital in shaping, refining, and READ MORE

On 24 May 2025, the Seventy-eighth World Health Assembly (WHA78) voted unanimously for the resolution “Rare diseases: a global health priority for equity and inclusion,” the first time the World Health Organization has formally elevated rare diseases to the top tier of its agenda. The text urges governments to integrate rare-disease policies in national health plans, expand newborn screening and timely diagnosis, and guarantee affordable access to medicines and assistive technologies under universal health coverage. It also encourages investment in digital tools, patient registries and centres of excellence, while promoting the active participation of patient organisations in policy design. Member READ MORE

A new questionnaire, “Promoting capacity-development actions” – has been released under one of the teams developing the European Rare Diseases Research Alliance (ERDERA) team that focuses on harmonization of the different national capacities. The goal is simple but urgent: to capture first-hand evidence of the funding gaps, technical hurdles and policy barriers that still keep many low- and middle-income or smaller European nations on the sidelines of global rare-disease science.