EURORDIS is pleased to announce that nominations for the EURORDIS Black Pearl Awards are now open until September, 4th. The Awards are presented to patient advocates and organisations, policy makers, scientists, companies, and media in recognition of their exceptional achievements and outstanding commitment to the rare disease cause. NOMINATIONS: Just like every year, nominations can be submitted for any individual, organisation or company (including yourself and/or […]
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As one of the outcomes of the MedTech for Rare Diseases Working Group led by IRDiRC members Anneliene Jonker and Marc Dooms, the Orphanet Journal of Rare Diseases has decided to focus a thematic issue on the questions regarding the research and development of orphan devices and medical technologies that are being developed for Rare Diseases […]
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London, UK – July 2022: The RDNN Board of Trustees and Management is delighted to announce the recent registration of RDNN as a registered UK charity. This is an important milestone as they strive forward on their mission to provide a network of specialist nurse support for every person with a rare disease. RDNN has […]
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We are proud to announce that the commentary on 10 years of progress and challenges of IRDiRC that was previously published in the prestigious journal Nature Reviews Drug Discovery is now fully available for reading. The article summarizes IRDiRC’s vision and goals and highlights its achievements and prospects after its first decade (2011–2021). The commentary […]
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Following the 11th European Conference on Rare Diseases and Orphan Products (ECRD) that was held from June 27th to July 1st and was organized by EURORDIS and co-organized by Orphanet with IRDiRC serving as an Associate Partner, the involved parties came together to write an open letter to Commissioner Kyriakides of the European Commission, calling for a […]
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With the occasion of the IRDiRC Joint Consortium Assembly – Scientific Committees Meeting, held on June 1st-2nd, 2022, in Paris, France, IRDiRC launched a series of interviews with its members on the importance of strong international collaboration in the rare disease community. We launch today the first video of the series with Dr. Christina Waters, […]
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IRDiRC has four Scientific Committees, one each for Diagnostics, Therapies, Interdisciplinary, and Regulatory aspects of rare diseases research. The Diagnostic Scientific Committee (DSC) identifies current and future bottlenecks to rare disease gene discovery, addresses challenges and roadblocks in rare disease diagnosis, and collaborates with international partners to develop tools and resources to facilitate genomic data discovery, analyses […]
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PARIS, FRANCE, JULY 5th, 2022: The International Rare Diseases Research Consortium (IRDiRC) is pleased to announce today the release of its Rare Diseases Research Initiatives State of Play 2019-2021 Report. This report is based on scientific articles and press releases published between 2019 and 2021; it seeks to inform stakeholders and the rare diseases community […]
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The National Institute of Neurological Disorders and Stroke (NINDS) recently created the Ultra-Rare Gene-Based Therapy (URGenT) Network. The URGenT Network supports the development of state-of-the-art gene-based therapies for ultra-rare neurological diseases, which affect as few or fewer than one in fifty thousand people. It supports Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) […]
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Rare Diseases International (RDI) would like to thank all who attended the RDI Informal Side-Event to the 75th World Health Assembly, which took place in Geneva, Switzerland, on May 24th, 2022. The event was opened by the World Health Organization and discussed how health systems around the world would be strengthened by the development of a Global Network for […]
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