The Foundation for Rare Diseases has developed a series of three online courses with European and international partners to better understand innovative therapies, health data, and translational research in the field of rare diseases. Developed with the support of a medical education grant from ASGCT and Pfizer, this course focuses on the scientific and translational […]
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We are pleased to share a new publication produced by the IRDiRC Functional Analysis Task Force, highlighting the growing impact of multi-omics in advancing rare disease research and precision medicine. Multi-omics, combined with advanced computational methodologies, integrates diverse omics datasets to deliver deeper insights into molecular interactions. This powerful approach has transformative potential for unraveling […]
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Undiagnosed Day 2026 is a two‑day, clinician‑led meeting in Gdansk (Poland) bringing together specialist clinicians, clinical geneticists and invited experts to support phenotype‑led diagnosis through live case discussion, shared clinical reasoning and practical exchange. The event is organised by the European Rare Diseases Research Alliance (ERDERA), the Wilhelm Foundation, the Medical University of Gdansk (GUMed), and the University Clinical […]
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IRDiRC Patient Advocates Constituent Committee (PACC) member, Genetic Alliance, is pleased to announce that 9 new clinical sites and 1 new laboratory joined the iHope program network, further strengthening the ability to reach families with rare genetic conditions. In addition, Dr. Jennifer Troyer, has joined the staff to support the continued growth and impact of […]
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The landscape of medical research is shifting: rare disease patient advocates are no longer just participants, they are the architects of discovery. Driven by an urgent need for life-saving treatments, these leaders are spearheading research initiatives with unprecedented momentum. To support this movement, Global Genes (IRDiRC Patient Advocates Constituent Committee member), in partnership with Boston […]
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The third Hope for Rare Science Conference will be held in Shanghai, China from June 25th to 27th, 2026. The conference is hosted by Hope for Rare Foundation, with National Children’s Medical Center/ Children’s Hospital of Fudan University and Chinese Organization for Rare Disorders (CORD) as co-hosts. The conference will last for 3 days, featuring […]
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The European Rare Disease Research Alliance (ERDERA) has re-opened its survey to better understand where the rare disease research community most needs external consultancy support with the goal to turn practical insights from researchers and stakeholders into targeted consultancy services that help projects overcome common bottlenecks and generate robust, timely evidence. Launched in September 2024 […]
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The CSNK2A1 Foundation Pilot Grant Program, developed in collaboration with the Orphan Disease Center University of Pennsylvania, member of the IRDiRC Funders Constituent Committee (FCC), will support one investigator-initiated research project focused on advancing the biological understanding of Okur-Chung Neurodevelopmental Syndrome (OCNDS) and accelerating therapeutic discovery. This pilot grant is designed to generate high-impact preliminary […]
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We are pleased to announce a new IRDiRC publication, “Challenges and opportunities for the use of telehealth in rare disease diagnosis, treatment, research, and education: key opinion leader interviews”, produced by the IRDiRC’s Enabling and Enhancing Telehealth for Rare Diseases Across the Globe Task Force, which explores how telehealth (TH) can transform diagnosis, care, research, […]
Read MoreIRDiRC is delighted to be represented by multiple members at the Undiagnosed Day 2026 taking place in Gdansk (Poland) and online. Bringing together specialist clinicians, clinical geneticists, and invited experts, this event aims to support phenotype-led diagnosis through live case discussions, shared clinical reasoning, and practical exchange. Inspired by the collaborative spirit of international Undiagnosed […]
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