Organized by the Rare Skin Diseases Network of the René Touraine Foundation, in association with ERN SKIN, the 1st World Congress on Rare Skin Diseases is an event that brings together the best experts, patient representatives and industry around the same cause: improving the care of patients with rare skin diseases. This in-person event will take place…
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Euronews has gathered a panel of experts, industry leaders and key players in Brussels to address the issue of how rare disease provision can be advanced in the European Union (EU) in a virtual debate entitled « Innovation in rare diseases: How can Europe be a global leader?« The virtual debate will take place on February 15th from 15.00 – 16.30…
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International Rare Diseases Research Consortium (IRDiRC) member Dr. Anneliene Jonker, Vice Chair of the Therapies Scientific Committee (TSC) of IRDiRC, has been interviewed extensively for an article published in the February 2022 issue of Medical Technology magazine. The article, titled “Medical devices for rare diseases: the unmet need”, presents the goals of the IRDiRC Working Group on MedTech for Rare Diseases, the…
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EU-STANDS4PM, a European standardization framework for data integration and data-driven in silico models for personalised medicine, has published a review article on computational models for clinical applications in the Journal of Personalized Medicine. In the article, the authors discuss the most relevant computational models for personalised medicine in detail that can be considered as best-practice…
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The European Joint Programme on Rare Diseases (EJP RD) is proud to announce that EJP RD member Prof. Franz Schaefer has been conferred the EURORDIS Scientific Award for his work and the positive impact he has made on rare disease research and the patient community. The EURORDIS Scientific Award, which forms part of the annual Black Pearl Awards, recognises…
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As part of the training activities proposed by EJP RD, an online training course on « Training on strategies to foster solutions of undiagnosed rare disease cases » is being organised by Istituto Superiore di Sanità (ISS) in close collaboration with EJP RD partners. The online event will take place over three days from on April 11th – 13th. Through…
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PARIS, France and TUCSON, Ariz., U.S., February 1st, 2022: The European Joint Programme on Rare Diseases (EJP RD) announces a collaboration with the Critical Path Institute (C-Path), an independent global nonprofit organization, to advance technologies and methodologies that are fit for regulatory purposes to further global rare disease research and drug development. C-Path has a proven…
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The European Medicines Agency’s (EMA) Clinical Trials Information System (CTIS) has gone live as of January 31st. CTIS is the backbone of the Clinical Trials Regulation that will harmonise the assessment and supervision of clinical trials in the European Union. The Clinical Trials Regulation and CTIS will strengthen clinical trials in the EU, ensuring better…
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The Therapies Scientific Committee of the International Rare Diseases Research Consortium (IRDiRC) is establishing a Task Force on Drug Repurposing Guidebook to help developers (of all kinds) navigating the rare disease landscape and identifying specific tools and practices of relevance for repurposing projects. The creation of the Development Guidebook will focus on repurposing approaches, following the same successful methodology used…
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The Dravet Syndrome Foundation Spain works to improve the quality of life of people with Dravet syndrome and their families. The foundation recently released its end-of-year 2021 Dravet Syndrome Therapeutic Pipeline Report. In this report, the Foundation undertook a comprehensive review of the approved and investigational drugs and therapies for Dravet syndrome. The report also…
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