There are over 6,000 different rare diseases identified to date that affect 3.5% – 5.9% of the worldwide population. They have a big impact not only on the person diagnosed, but also on their families, friends, care takers and society as a whole. This is why development of medicines addressing rare diseases, also known as […]
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The Canadian Institute of Health Research – Institute of Genetics is pleased to announce the upcoming funding opportunity “National Pediatric Rare Disease Clinical Trials and Treatment Network”. The specific objectives of this funding opportunity are to: Develop a platform to support pediatric rare disease clinical trials in Canada; Increase the capacity to perform rare disease […]
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IRDiRC’s Diagnostic Scientific Committee (DSC) plays a critical role in identifying and addressing current and future bottlenecks to rare disease gene discovery and diagnosis. As we work towards improving rare disease diagnosis globally, the DSC is currently seeking to expand its membership by welcoming a rare disease diagnostic expert from Africa or Latin America and a […]
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The 35th Annual Meeting of EMSOS will take place this year on 10-12 May, 2023, in Brussels (Belgium), and it will join the efforts of all Belgian University Hospitals and professions represented in the organizing committee to address this year’s theme “Back to (multidisciplinary) reality: how far do we go?” Marc Dooms, IRDiRC Interdisciplinary Scientific […]
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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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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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