Members of the global rare disease community gather in Barcelona for hands-on training
Earlier this month, Barcelona hosted the 2025 edition of the EURORDIS Open Academy Schools, bringing together 77 patient advocates and early-career researchers from 27 countries for three days of practical learning and exchange. Held from 2 to 4 June, the event marked the first edition of the Schools delivered as part of the ERDERA partnership, and reaffirmed the shared goal of strengthening the community of people driving progress in rare-disease research. The programme combined classroom sessions with immersive field visits, offering a rich learning experience. Participants explored cutting-edge facilities at Hospital Sant Joan de Déu, including the CORTEX command centre, READ MORE
Triplet repeat expansion project sets benchmark for public-private collaboration
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 disease research. Led by Professor Robert S. Lahue of the University of Galway, this preclinical project focused on triplet repeat expansion (TRE) diseases – a category of over 30 inherited neurological disorders caused by unstable DNA repeats. These diseases are difficult to treat, making this an important area for development of new tools to expedite drug discovery.
“There’s a lot of value in connecting with other countries — learning what has worked, what hasn’t, and applying those lessons”
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 together policymakers, clinicians, academics and—crucially—patient advocates to coordinate Ireland’s national efforts and align them with wider European initiatives such as ERDERA. The group aims to streamline collaboration, co-ordinate resources and ensure that patients’ voices shape both implementation of the forthcoming national rare-disease strategy (to 2030) and Ireland’s engagement in international research and policy-making.
“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”
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 creation in 2021. Under her guidance, this well-established NMG links policymakers, clinicians, scientists and patient organisations, channeling Portuguese priorities into European initiatives such as the European Rare Diseases Research Alliance (ERDERA). The group now plays a central role in aligning national efforts with the newly launched “Plano de Ação para as Doenças Raras 2025-2030”, helping Portugal make the most of international collaboration while improving care and research for people living with rare READ MORE
ERDERA diagnostics research task force showcases impact and ambition at ESHG 2025
The European Human Genetics Conference 2025 (ESHG) provided a significant platform for the ERDERA Diagnostics Research task force to share its strategic vision, scientific progress, and commitment to innovation in the field of rare disease diagnostics.
Europe’s rare-disease alliance charts next steps at Riga workshop
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 entitled “How to maximise the power of national plans for rare diseases and strengthen their capacity to foster rare-disease research.” The event, which coincides with the close of ERDERA’s first year, focused on strengthening the alignment between national action plans and the wider European research agenda while accelerating the roll-out of National Mirror Groups, or NMGs.
ERDERA’s Networking Support Scheme opens to forge new rare disease and rare cancer alliances
The European Rare Disease Research Alliance (ERDERA) has launched its Networking Support Scheme (NSS), a continuously open call designed to knit together researchers, clinicians and patient advocates working on rare diseases and rare cancers. Proposals may be lodged at any time, but the first collection round will be assessed immediately after the 7 October 2025 deadline, allowing successful teams to start planning events without delay. Under the scheme, applicants can request up to €30 000 per networking event to cover meeting costs, travel costs, hybrid-meeting platforms and other essentials. Rounds will be held every six months until April 2029—or until READ MORE
ERDERA launches survey to put rare-disease patients at the heart of research
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
World Health Assembly adopts first-ever resolution on rare diseases, signalling a new era of global collaboration
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
ERDERA has received funding from the European Union’s Horizon Europe research and innovation programme under grant agreement N°101156595.
Views and opinions expressed are those of the author(s) only and do not necessarily reflect those of the European Union or any other granting authority, who cannot be held responsible for them.
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ERDERA is coordinated by Insem