IRDiRC is proud to announce that, as one of the outcomes of the MedTech for Rare Diseases Working Group led by IRDiRC members Anneliene Jonker and Marc Dooms, the Orphanet Journal of Rare Diseases has decided to focus a thematic issue on the questions regarding the research and development of orphan devices and medical technologies that…
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 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 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,…
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…
EJP RD’s innovative Rare Diseases Research (RDR) Challenges call in partnership with Fondation Maladies Rares was aimed at facilitating and funding collaboration between industry, academia, SMEs, and patient organisations to solve specific research challenges in rare diseases. The second challenge issued under the RDR call was for the development of a delivery system for intranasal administration of biological drugs to neonates. We are glad…