IRDiRC 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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Global Genes is excited to share the news that the Xcelerate RARE Open Science Data Challenge has officially opened! The Xcelerate RARE: A Rare Disease Open Science Data Challenge is bringing together researchers and data scientists in a collaborative and competitive environment to make the best use of patient-provided data to solve big unknowns in […]
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This year the annual conference of the International Mouse Phenotyping Consortium (IMPC) will be held at Keble College, Oxford, UK from the 10-11 July, 2023. The conference is entitled: “The Power of Mouse Genetics: Opportunities for Genomic and Precision Medicine“. It features an outstanding list of distinguished speakers at the forefront of mouse and human […]
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The Solve-RD consortium and associated European Solve-RD network, including among others six European Reference Networks, EURORDIS, Orphanet and leading European Rare Disease Clinicians and Researchers in 20 countries, call upon all European RD stakeholders, including EU Member States, the European Commission, the Council of the European Union, the general public and private organisations active in […]
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On March 22, 2023, the Canadian Federal Government announced the launch of Canada’s Rare Disease Drug Strategy with a $1.5 billion commitment over three years to “improve access to new and emerging drugs, as well as support enhanced access to existing drugs, early diagnosis, and screening for rare diseases, and to improve the collection and […]
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A new network for nurses has been created to exchange skills, knowledge, solve problems and generate new ideas for the rare disease community. The first meeting to develop the network was a Roundtable event held on March 9-10, 2023, in Singapore, and was attended by rare disease global leaders and 30 nurses from 25 countries. […]
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The Public Symposium is open to the entire human genetics and rare disease community in Europe and beyond. In addition to presentations on Solve-RD achievements, we are very happy to have two keynote speakers: Ines Thiele (University of Galway) will outline the way “Towards a personalised systems biomedical approach for the diagnosis of inherited metabolic […]
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On May 2 and 3, 2023, the FDA’s Center for Drug Evaluation and Research (CDER) and the Johns Hopkins University’s Center of Excellence in Regulatory Science and Innovation (JHU CERSI) will host a jointly sponsored virtual workshop on addressing challenges in the design and analysis of rare disease clinical trials. This workshop will discuss: How […]
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The World Health Organization (WHO) is seeking experts to serve as members of the Strategic Advisory Group of Experts on In Vitro Diagnostics (SAGE IVD). This “Call for Experts” provides information about the advisory group in question, the expert profiles being sought, the process to express interest, and the process of selection. For more information […]
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Prof. Guillaume Canaud (Necker Hospital for Children, AP-HP, France) offered a great presentation on “Targeted therapy for patients with PIK3CA-related overgrowth spectrum“ at the RE(ACT) Congress in Berlin (Germany). Check out his impressions on the IRDiRC Conference x RE(ACT) Congress at the following link: here.
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