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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The rare disease community will convene in Amsterdam this October for the World Orphan Drug Congress Europe 2026, bringing together stakeholders from across the orphan drug and rare disease ecosystem. The […]
Read MoreIRDiRC is pleased to announce the appointment of 3 new members to the Therapies Scientific Committee (TSC): The Therapies Scientific Committee brings together international experts committed to advancing therapeutic development […]
Read MoreIRDiRC is delighted to announce the appointment of new members to the Regulatory Scientific Committee (RSC). Established in 2022, the Regulatory Scientific Committee (RSC) was created to address regulatory gaps […]
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