Hosted by the BLACKSWAN Foundation, the International Rare Diseases Research Consortium (IRDiRC) and the European Rare Diseases Research Alliance (ERDERA), the RE(ACT) Congress & IRDiRC Conference offers a unique opportunity to connect face-to-face with our community and take part in a truly collaborative environment. Top Highlights: – Participate in cutting-edge research and policy discussions– Network […]
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AFM-Téléthon is launching its 2025 calls for proposals to support international research on neuromuscular disorders and rare genetic diseases. These calls are open to both French and international teams, offering funding opportunities for: ? Fundamental research and physiopathology projects.? Development of innovative therapeutic approaches.? Projects aimed at improving patient care and quality of life measures. […]
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New funding opportunity from the US National Institutes of Health (NIH) – PAR-25-122: Pilot Projects Investigating Understudied Proteins Associated with Rare Diseases (R03 Clinical Trial Not Allowed) The purpose of this notice of funding opportunity (NOFO) is to solicit applications for pilot projects to elucidate a role for understudied proteins in rare disease. Awards will support […]
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Exciting news from the IRDiRC PACC member EURORDIS! The Rare Barometer survey on the impact of rare diseases on daily life is now open. Open worldwide and available in 25 languages, from 10 July to 8 September. More information about the survey: Where can I find the survey and in how many languages is it […]
Read More“Earlier, faster and more accurate diagnosis” We are delighted to share the results of the the recent Rare Barometer survey, developed by the IRDiRC PACC member organization EURORDIS-Rare Diseases Europe, which shows that the average rare disease patient in Europe waits nearly five years for a diagnosis. Check out more details about this survey’s findings […]
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IRDiRC announces the launch of a new paper on “Newborn screening in Mexico and Latin America: present and future”, part of the Newborn Screening Initiative Real-World Applications and Technologies special edition, now fully available in the Rare Disease and Orphan Drugs Journal. “The first newborn screening program (NBS) to be implemented in Latin America was […]
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The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal year (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 support of a […]
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Despite advancements, most rare diseases lack effective therapies, underscoring the necessity for a strategic overhaul in the approach to research and development. To address the multifaceted challenges in rare disease research, a unified framework that encompasses the recommendations across clinical trials, diagnostics, and translational research is proposed by the Rare Disease Moonshot. Central to this […]
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A new IRDiRC paper on “Global health for rare diseases through primary care”, the work of the IRDiRC Primary Care Task Force, is now available open access in the The Lancet Global Health: https://lnkd.in/e_6_Rczr. The Task Force brought together a multi-stakeholder group of experts to assess the priority research areas in primary care. “Family medicine […]
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The European Joint Programme on Rare Diseases is organizing and End-User Training intended for potential users of the Virtual Platform (VP) that is developed in the context of the EJP RD Pillar2 activities. At the end of this webinar, you will be able to: Describe how to interact with the Virtual Platform as a […]
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