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—seeks first-hand assessments of what works, and what falls short, when patients are invited to shape studies that ultimately affect their […]
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The FDA’s Office of Orphan Products Development (OOPD) is excited to announce a funding opportunity for fiscal year 2026 aimed at advancing natural history studies in rare diseases and conditions: “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)” (https://grants.nih.gov/grants/guide/rfa-files/RFA-FD-25-020.html). Receipt Date: October 21, 2025 FOA Number: RFA-FD-25-020 The purpose of this […]
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Global Genes, IRDiRC Patient Advocacy Constituent Committee member organizes its 2025 RARE Drug Development Symposium, being held on 3-4 September, 2025 in Boston (MA, USA). This hands-on, expert-led event is designed to help rare disease advocates build expertise, connect with leading therapy developers and researchers, and confidently navigate the drug development process. Whether you’re initiating […]
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The Wiedemann-Steiner Syndrome Foundation, in partnership with the Orphan Disease Center University of Pennsylvania, is proud to announce three new 2-year research grants to support scientific advancement in Wiedemann-Steiner Syndrome (WSS). WSS is a rare genetic disorder caused by mutations in the KMT2A (MLL) gene, impacting gene expression and leading to significant cognitive and behavioral […]
Read MoreThe National Center for Advancing Translational Sciences (NCATS) is seeking input from the scientific and research community on the development of advanced tools to predict the toxicity of oligonucleotide therapeutics, a critical step toward accelerating the safe and ethical advancement of these promising treatments. Oligonucleotide therapeutics (e.g., ASOs, siRNAs, miRNA mimics) hold major potential for […]
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IRDiRC Diagnostic Scientific Committee (DSC) unites clinicians and specialists in genetics, genomics, bioinformatics, molecular diagnostics, and biochemistry to help reduce the diagnostic odyssey for the patients living with rare diseases. DSC plays a key role in identifying the barriers related to gene discovery and diagnosis and actively works to overcome these challenges by developing strategies to […]
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The World Orphan Drug Congress Europe is the largest and most established event globally for orphan drugs and rare diseases. With over 150 speakers already confirmed, WODC Europe connects global experts, industry leaders in the orphan drug space, and patient advocates, that drive transformative change in the rare diseases ecosystem. We are pleased to announce […]
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IRDiRC is proud to announce the publication of the latest article in Therapeutic Advances in Rare Disease: “From Roadmap to a Sustainable End-to-End Individualized Therapy Pathway.” This publication builds on the foundational work of the IRDiRC N-of-1 Task Force, expanding from the initial roadmap to explore how N-of-1 therapies—custom treatments for individuals with ultra-rare genetic conditions—can […]
Read MoreIRDiRC has four Scientific Committees: Diagnostics, Therapies, Interdisciplinary, Regulatory. The Interdisciplinary Scientific Committee (ISC) is a multi-stakeholder, multi-disciplinary group of experts working on rare disease (RD) data sharing, ontologies, natural history, biobanks, registries, ethical and societal aspects of rare diseases. ISC would like to broaden the committee membership, to represent the worldwide rare diseases community in its […]
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At the 78th World Health Assembly, World Health Organisation Member States have officially adopted the first-ever Resolution on Rare Diseases — a landmark step toward greater equity and recognition for the 300 million people affected worldwide. IRDiRC is proud to have played a role in this global achievement. This milestone reflects the strength of international […]
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