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, Middle Eastern & African Society for Biopreservation and Biobanking (ESBB) and BBMRI-ERIC are organising the Europe Biobank Week Roadshow 2022 (EBW22) in partnership with the BBMRI ERIC Stakeholder Forum Patient Pillar, BBMRI.it, the Research Translational (IRCCS) Paediatric Bambino Gesù Hospital, and the paediatric network IDEA. The second stop of the EBW22 will be…
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…
The pre-announcement of the CHIST-ERA Call ORD is online! It will target R&D projects in the following topic: « Open & Re-usable Research Data & Software« . This one-step submission and evaluation call tackles the challenge of open research data and software from the perspective of their possible reuse. The objective is to create the conditions for…
The Duchenne Data Foundation is pleased to invite you to the 6th edition of the Duchenne Patient Academy, of which the theme is « Changing Perspectives ». The conference aims to inspire and equip patient advocates to become change makers and build a better future for people living with dystrophinopathies. The Academy provides online training for Duchenne…
A funded networking event, the first Argonaute Syndrome Science & Family conference, is being organized to bring together patient families, researchers and clinicians of the AGO1 and AGO2 communities to facilitate the sharing of knowledge. This event has received funding support from the EJP RD’s Networking Support Scheme (NSS) funding opportunity. The event will be held…
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,…
As part of the training activities proposed by the EJP RD, a 3-day training course titled “Quality assurance, variant interpretation and data management in the NGS diagnostics era” is being organised by Universitätsklinikum Tübingen, in close collaboration with EJP-RD task partners, aimed the international research community: clinicians, medical specialist, laboratory scientists (EBMG-registered), junior laboratory scientists, clinical geneticists, policy…