We are thrilled to announce the launch of the European Joint Programme on Rare Diseases (EJP RD) survey aimed at assessing the knowledge and impact of Rare Diseases research (data) resources and tools developed within the project. As a member of the Rare Diseases research community, your input is invaluable. This survey will help in […]
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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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A new publication about incorporating Spinal Muscular Atrophy (SMA) in newborn screening programs across Europe is now available in the Rare Disease and Orphan Drugs Journal. The paper highlights how patient advocacy organizations represent a key partner in ensuring equitable and early diagnosis. đź“– Read the full publication here: https://lnkd.in/emYepUDXđź“š More publications on Newborn Screening: […]
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The European Commission has published a new Guidance on clinical evaluation of orphan medical devices on June 26th. Orphan devices are medical devices or their accessories, which are intended to be used for diseases or conditions affecting only a small number of individuals each year. Often, they are used to treat a rare disease or medical […]
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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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Listen now to EURORDIS RARE ON AIR latest episode, where Julien Poulain (Communication Manager at EURORDIS) speaks with Ayça Ĺžahin, a PhD student in neuroscience living in Turkey with Spinal Muscular Atrophy (SMA), who shares her and her brother’s journey to receiving an SMA diagnosis. Ayça highlights the critical importance of early and accurate diagnoses […]
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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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