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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Rare Diseases International (RDI) will participate to the WHO Expert Committee on the Selection and Use of Essential Medicines Open Session on 24 April 2023, and provide a statement regarding essential medicines for rare diseases. The agenda of the event is available here. More information: https://www.who.int/groups/expert-committee-on-selection-and-use-of-essential-medicines/24th-eml-expert-committee
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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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This year’s conference theme is “Duchenne Care: 5 Years After the 2018 Care Considerations (Lancet)”, and will take place online on June 7 and 8, 2023, from 15:00 – 18:00 CEST. Particularly, the conference will include new publications, information about the implementation of these Care Considerations in different countries. Top experts in the field will […]
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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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As part of the NIH’s Somatic Cell Genome Editing (SCGE) program, the Office of Strategic Coordination (Common Fund) intends to publish a prize competition announcement to solicit entries for the multi-phase TARGETED (Targeted Genome Editor Delivery) Challenge. The TARGETED Challenge aims to improve the current state of in vivo delivery technologies for genome editors in two […]
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The next installment of the “Challenges in Pediatric Neuroscience Research Webinar Series”, will be on May 12th, 2023 from 2-3 PM EST. In this episode of our ongoing series, we will be speaking with Dr. Heather Hazlett, a pediatric neuropsychologist at the University of North Carolina at Chapel Hill, about remote neuropsychological assessments. This type […]
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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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