We are delighted to share Together4RD’s position statement, which delves into a critical topic: the untapped potential of European Reference Networks (ERNs) in addressing the unmet needs of people living with a rare disease.Find the publication here
Under the Horizon Europe Framework Programme, a new two-stage call for proposals for « Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases » was published on 27 July, 2023. The project generated from the topic should not only develop capacities and capabilities to execute innovative trial designs, but also plan to identify solutions…
The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal years (FY) 2023 – FY 2025 to support clinical trials for rare diseases and conditions. The purpose of this funding opportunity announcement is to fund clinical trials of products evaluating efficacy and/or safety in…
The ERICA & EJP RD Joint Conference is scheduled for November 21st, 2023, in Amsterdam. This event aims to unite stakeholders in European Reference Networks (ERNs) and Rare Disease (RD) Research. It will bring together medical professionals, researchers, patient advocates, policymakers, and industry representatives interested in improving care and advancing research for rare diseases. A…
The hereditary tumour syndrome that correlates with a carriership of a pathogenic CDH1 variant is very rare and as such underrecognized. Early recognition however can save lives by preventing death due to diffuse type gastric cancer at early age. Therefore, (refresher) training of all clinical workers involved in early recognition, surveillance, treatment, and aftercare is important. This two-days…
ECRIN recently launched the applications for an upcoming training series for investigators and project manager in our ECRIN countries to support the submission of proposals for clinical studies to Horizon Europe. Everything is explained in full here.
The Rare Diseases (RD) Moonshot was set up to boost public private collaborations in the areas where there are with no treatment options and there is no R&D going on. Modernising clinical trials (design, conduct, regulatory sciences) to make them suitable for very small populations was identified by the RD Moonshot partners as one of…
IRDiRC proudly announces the publication of the manuscript “Targeting shared molecular etiologies to accelerate drug development for rare diseases“ online, today. The manuscript is published in the EMBO Molecular Medicine Journal and freely accessible online here.
The MSCA-DN-2023 Call info event that took place on 14 June 2023, with more than 1000 registrations, around 1500 viewers and 1600 people actively replying to polls, the overall participation rates are impressive and speak for the big success of the event. We received in total 187 questions through slido; 41 were replied live…
The abstract on “The European Joint Programme on Rare Diseases: an integrative approach fostering the RD research ecosystem“ submitted in the context of the European Conference on Rare Diseases (ECRD) 2022 in which EJP RD was serving as a full partner, was published in the Orphanet Journal of Rare Diseases on Friday, 2nd June (under P43 section).…