On 15 October, EURORDIS-Rare Diseases Europe launched “A European Blueprint for Rare Diseases”, setting out the rare disease community’s proposals for the priorities and implementation of a future EU Action […]
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IRDiRC is pleased to announce the release of a new publication in the Orphanet Journal of Rare Diseases, “Unveiling the depth of the knowledge gap in the Universe of Rare […]
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On behalf of Rare Diseases International (RDI), member of IRDiRC Patient Advocates Constituent Committee, we are delighted to announce the opening of the RDI Youth Leadership Programme 2027–2028. Over 2 […]
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Rare diseases often face significant challenges in drug discovery and development, including limited patient populations, fragmented data, and high research costs. A recent editorial in Drug Discovery Today highlights the potential of […]
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The International Rare Diseases Research Consortium announces two new members for its Diagnostics Scientific Committee (DSC). The Diagnostics Scientific Committee brings together international experts in clinical genetics, genomics, bioinformatics, molecular […]
Read MoreThe International Rare Diseases Research Consortium (IRDiRC) Working Group on the Engagement of Young People Living with Rare Diseases in Therapy Development has published a perspective article in Frontiers in Pediatrics entitled “Engaging Children, Adolescents and Young Adults Living with Rare Diseases as Research Partners: Practical Considerations for Meaningful […]
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What can happen when researchers, clinicians, patient advocates, policymakers, industry experts, and emerging voices come together to advance rare disease research? From 10–12 March 2027, the rare disease community will […]
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The U.S. FDA has approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment specifically approved for pediatric patients with Sanfilippo syndrome type A (MPS IIIA), a rare and progressive genetic disorder affecting […]
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Beacon for Rare Diseases has launched Rare Insights – a ground-breaking study exploring the value and impact of rare disease patient groups, alongside the challenges they face. We need your […]
Read MoreThe International Rare Diseases Research Consortium is pleased to welcome to its Interdisciplinary Scientific Committee (ISC) two new members: Elizabeth Hart – Associate Director of Rare Disease (Acting), Center for […]
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