As part of the training activities proposed by EJP RD, an 3-day 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 training will be held in Rome, Italy on 13-15 March 2023. Registration is open until 14 January 2024.…
The French Foundation for Rare Diseases is delighted to announce the upcoming opening of a new facilitation window of the MOOC “Diagnosis Rare Diseases: from the clinic to research and back” on September 19th. This update will be available until December 3rd 2022. During this period, expert mentors will be actively present on the platform to answer the…
Rare Diseases International is delighted to announce their upcoming webinar on the use of the International Classification of Diseases 11th Revision (ICD -11) for rare diseases, which will take place on September 27th, at 14.00 CET. The webinar will be co-hosted by RDI and the World Health Organization. The ICD is developed and updated by the…
The EJP RD « Bring Your Own Omics Data » Workshop is now open. The 2-day workshop will take place on November 17th and 18th 2022, face to face in Nijmegen (Netherlands). Costs for travel and accommodation might be available for participants from ERNs and EJP RD beneficiaries. The « Bring Your Own Omics Data » (BYOOD) Workshop aims to help with optimising and integrating…
The EJP RD is proud to announce that the Smart Guidance tool for the FAIRification of rare disease registries was released on July 18th. The questionnaire-based Smart Guidance tool is built on the Data Stewardship Wizard; it will guide users (e.g., data stewards) through the process of making their registry more FAIR. It covers various…
The International Rare Diseases Research Consortium (IRDiRC) is proud to announce that the commentary on 10 years of progress and challenges of IRDiRC that was previously published in the prestigious journalNature 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…
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…
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 EJP RD serving as a Full Partner, the involved parties came together to write an open letter to Commissioner Kyriakides of the European Commission, calling for…
The European Medicines Agency (EMA) has recommended a conditional marketing authorisation in the European Union (EU) for Tecvayli (teclistamab), a monoclonal antibody that targets BCMA and CD3 proteins, for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an…
A new, free-of-charge webinar in collaboration with the AAAS/Science magazine is available on demand since July 9th. This webinar broadcasted on July, 7th discusses how the standard model for developing new medicines is often not profitable enough for pharmaceutical companies to produce therapeutics for the thousands of rare diseases known to exist. In the past,…