IRDiRC is delighted to share the manuscript “How to START? Four pillars to optimally begin your orphan drug development“, developed by the members of the IRDiRC Task Force “Orphan Drug Development Guidebook (ODDG)” together with the members of the Therapies Scientific Committee (TSC). The paper proposes a checklist for orphan drugs named START – a […]
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Don’t miss out the European Haemophilia Consortium (EHC) Conference that will take place this year on 6-8 October, 2023, in Zagreb, Croatia, where IRDiRC Interdisciplinary Scientific Committee (ISC) Vice Chair, Marc Dooms (University Hospitals Leuven, Belgium), will present at the European Rare and Inhibitor Network (ERIN) session: “Basket trails – changes that we need”. Check […]
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Rare disease research presents unique challenges due to the small size of the populations of people living with these conditions. Collecting and curating large study cohorts in rare diseases presents a real problem, which can limit understanding of the clinical spectrum of disease. The SANOFI Rare Disease Registries represent more than 30 years of real-world […]
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IRDiRC is delighted to present you Karla Ruiz de Castilla, PACC member representative of the Iberoamerican Alliance for Rare Diseases (ALIBER). Karla is also the President of ESPERANTRA, a non-profit organization founded with the purpose of contributing to the reduction of mortality from cancer, chronic and rare diseases, with a strong advocacy focus on equal […]
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The Rare Disease (RD) Moonshot steering group has just published its recommendations for research funders and health research players from the industry and public sector on research needs that can be best addressed by public-private collaborations to optimise clinical trials in small populations. The RD Moonshot was set up to boost public private collaborations in the […]
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Inaugurated in 2012, China Rare Disease Summit has become the largest and the most comprehensive rare disease conference in China. The annual summit promotes open communications among multiple stakeholders, fosters collaboration, and makes significant contributions to policy advocacy in China. The largest and the most comprehensive rare disease conference in China The most robust platform […]
Read MoreIRDiRC proudly announces the publication of the manuscript “Targeting shared molecular etiologies to accelerate drug development for rare diseases“ online, today. The manuscript is published in the EMBO Molecular Medicine Journal and freely accessible online at the following link: https://www.embopress.org/doi/full/10.15252/emmm.202217159.
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The Innovative Health Initiative (IHI) Program Office just pre-published the call for proposals on “Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases”. Check out additional details at the following link: https://www.ihi.europa.eu/sites/default/files/uploads/Documents/Calls/FutureTopics/DraftTopic_TrialsRareDiseases_June2023.pdf Don’t miss out the recording from the information webinar: here. The topics will be formally published at the end of July […]
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The International Rare Diseases Research Consortium (IRDiRC) announces new leadership changes in its Funders Constituent Committee (FCC). Dr. Adam Hartman (Program Director at the National Institute of Neurological Disorders and Stroke (NINDS), USA), FCC Chair, and Dr. Catherine Nguyen (Director at the Thematic Institute of Genetics, Genomics, and Bioinformatics, INSERM, France), FCC Vice Chair, have […]
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This call builds on World Health Organization’s enduring efforts in the field of Genomics and follows the publication of the Report “Accelerating access to genomics for global health: promotion, implementation, collaboration, and ethical, legal and social issues” by the WHO Science Council in 2022. The WHO is now seeking to establish a Technical Advisory Group […]
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