Fondazione Telethon announced that it has submitted the Marketing Authorization Application (MAA) for the gene therapy – etuvetidigene autotemcel – for the treatment of patients with Wiskott-Aldrich Syndrome (WAS), a rare genetic disease of the immune system to the European Medicines Agency (EMA). If approved, etuvetidigene autotemcel will provide a valid treatment option, with excellent results in terms of both safety […]
Read MoreThe call for members for one Working Group and four new Task Forces is ongoing! The activities planned for 2025 are: (1) Task Force on “Preventive Medicines”: https://lnkd.in/eb4p-NSJPlease add in the subject of your email the reference for this call Ref: TF-PM. (2) Working Group on “Young Patient Involvement in Therapy Development”: https://lnkd.in/eTnneeZePlease add in the […]
Read MoreWe are pleased to share that the webinar series organized by Together for Rare Diseases & ERICA, brought together over 150+ members of the rare disease research community, who had the chance to learn about the contributions and the practicalities of public-private partnerships between ERNs and pharmaceutical companies. With panellists from European Reference Networks, pharmaceutical companies […]
Read MoreWe are pleased to announce that the RE(ACT) Congress and IRDiRC Conference 2025 have extended the regular registration and abstract submission deadline until the end of January. Taking place from 5–7 March 2025 in Brussels, this milestone event marks a significant moment for IRDiRC and the global rare disease research community. Join us for this […]
Read MoreThe International Rare Diseases Research Consortium is launching a call for experts to support five new activities. Check out the newly approved Task Forces and Working Group : More details on the experts’ profiles: here More details on the experts’ profiles: here More details on the experts’ profiles: here More details on the experts’ profiles: […]
Read MoreThe Interdisciplinary Scientific Committee (ISC) and Patient Advocacy Constituent Committee (PACC) are establishinga Task Force on “Stigma and rare diseases”. Globally more than 300 million people are living with a rare disease (PLWRD). For many, stigma is a critical health and social barrier with a high unmet need, that is estimated to be an overtly expressed […]
Read MoreThe Therapies Scientific Committee (TSC) is establishinga Working Group on the Engagement of Young People Living with Rare Diseases in Therapy Development. The aim of the working group is to develop a landscape view of the field and create practical tools such a decision tree to help inform stakeholders involved in pediatric rare diseases research and therapies development. Engaging […]
Read MoreThe Funders Constituent Committee (FCC), the Companies Constituent Committee (CCC) and the Regulatory Scientific Committee (RSC) are establishing a Task Force on International Regulatory Convergence aiming to identify barriers towards mutual recognition of the approval of rare disease therapies by different jurisdictions and propose concrete solutions. The Task Force will follow a disease agnostic approach and focus […]
Read MoreThe Diagnostic Scientific Committee (DSC), Interdisciplinary Scientific Committee (ISC), and Therapies Scientific Committee (TSC) are jointly establishing a Task Force on “Bridging Diagnostics to Therapies and Care” with the following objectives: We are looking for members to populate this Task Force with the below expertise in one or more of the following areas: The usual time commitment includes […]
Read MoreThe Therapies Scientific Committee (TSC) is establishing a Task Force on Preventive Medicines, where drug activity can either halt or suppress the onset of disease symptoms or the condition itself. Across different stakeholders, there is a growing consensus that prevention must become a more significant part of health policy and pharmaceutical development programmes. The aim of the Task […]
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