Are you advancing research, shaping policy, or driving advocacy for people living with rare diseases? Share your work with Europe’s rare disease community at the European Conference on Rare Diseases & Orphan Products (ECRD 2026) — Europe’s largest, patient-led rare disease event organized by EURORDIS Rare Disease Europe, taking place on 3–4 June 2026 in Prague, Czech Republic. […]
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The World Orphan Drug Congress 2025 brought together leading voices in the rare disease community for 3 days of insight, collaboration, and forward-looking discussion. Representatives from the International Rare Diseases Research Consortium (IRDiRC) shared key perspectives on advancing research and improving access to therapies for people living with rare conditions. A highlight of the congress, which took place […]
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Regulatory sandboxes are emerging as a powerful tool to accelerate innovation in healthcare, and their potential in the rare disease space is especially promising as they offer flexible, structured environments where novel approaches to regulation can be safely tested and refined. This topic was part of the special session at the IRDiRC Consortium Assembly and […]
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IRDiRC is pleased to share a new publication “What Matters Ethically About How the UDN Has Changed Since Its Inception”, recently published in AMA Journal of Ethics. For individuals living with undiagnosed rare diseases, the search for a diagnosis can be long, uncertain, and emotionally burdensome. A delayed diagnosis, or none at all, can significantly add […]
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IRDiRC announces publication of a new manuscript, developed as part of the International Rare Disease Research Consortium (IRDiRC) N-of-1 Task Force: “Applying the IRDiRC N-of-1 Therapy Task Force Eligibility Criteria for Individualised Therapies – Use Case: Duchenne Muscular Dystrophy“. Nucleic acid-based therapies, including antisense oligonucleotides (ASOs), are opening new doors for the treatment of rare diseases. With over […]
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The 2nd International Conference on Clinical Research Networks for Rare Diseases will take place on 9–10 December 2025 in Heidelberg, Germany, with full hybrid access for participants worldwide. This edition features a distinctly hands-on format, placing delivery at the centre. Each session —plenary and workshops alike— will close with agreed action points that will be captured live […]
Read MoreThe International Rare Diseases Research Consortium (IRDiRC) is pleased to participate in this year’s World Orphan Drug Congress Europe, taking place from 27–29 October 2025 in Amsterdam, Netherlands. We warmly invite all attendees to visit the IRDiRC booth (10.510) to learn more about our mission, collaborative initiatives, and how we are accelerating the development of diagnostics and therapies for people living with […]
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Professor Sally Ann Lynch, chair of IRDiRC Diagnostics Scientific Committee (DSC), will be chairing the upcoming ERN ITHACA webinar on “Genetics and unique populations: The Case of the Finnish, Roma and Irish Travellers”.With a deep expertise in rare diseases, endogamous populations and clinical risk, Professor Lynch will discuss about different barriers in healthcare access across […]
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IRDiRC Funders Constituent Committee (FCC) Member Fondazione Telethon is pleased to announce a new discovery from San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), which reveals the key liver cells driving organ growth and advancing pediatric gene therapy. Published in the Journal of Hepatology, the study reveals that a minority of liver cells in newborns […]
Read MoreIRDiRC members Marjon Pasmooij (Head of the Science Department at the Dutch Medicines Evaluation Board, and member of Therapies Scientific Committee), Mary Wang (Director Patient Engagement at Genespire, and member of Interdisciplinary Scientific Committee) and scientific project manager Alexandra Tataru participated in the Multi-Stakeholder Workshop on Prioritization of Rare Diseases for ATMP Development, organized by […]
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