The European project ERDERA has opened a call to select patients with a rare disease to participate in a training course organized by TEDDY Network with the support of c4c-S, IMAGINE, and EURORDIS, from 2-3 May 2025, in Athens, Greece. The training will provide tailored educational content in English, including youth-friendly resources, videos, serious games, webinars, and […]
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IRDiRC is delighted to present the outcome of the collaboration between the European Federation of Pharmaceutical Industries and Associations (EFPIA), the Rare Disease Moonshot and the IRDiRC Companies Constituent Committee: “The different contributions of the industry in Public-Private Partnerships in Rare Diseases Research continuum” report, which marks a pivotal moment in research advancement, innovation and […]
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We are delighted to announce that IRDiRC Chair, Dr. David Pearce, will be speaking at the World Orphan Drug Congress USA this April, in Boston, MA, USA. The World Orphan Drug Congress brings together leading pharmaceutical and biotech companies, government and regulatory bodies, patient advocacy organizations, payers, and investors. The congress serves as a platform […]
Read MoreWe are excited to announce the release of our latest scientific article: “Drug–device combinations in rare diseases: Challenges and opportunities” published in Drug Discovery Today, co-authored by members of Therapies & Interdisciplinary Scientific Committees. Why does this matter? Drug–device combinations (DDCs) offer a groundbreaking approach to treating rare diseases by enhancing drug delivery, improving targeting, and minimizing […]
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The Jack Bear Foundation, in partnership with the Orphan Disease Center, University of Pennsylvania, is now accepting applications for the 2025 Grant Program to support critical research on SCAR-15, a rare degenerative genetic disorder linked to mutations in the RUBCN gene. This grant will provide up to $100,000 for innovative research aimed at advancing our […]
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An upcoming ISPOR Webinar will explore the challenges in evaluating rare, pediatric, degenerative diseases by sharing the example of Duchenne Muscular Dystrophy (DMD) as a case study. Experts will dive into the complexities of traditional HTA frameworks and share innovative insights on assessing orphan drugs. This is a unique opportunity to gain valuable knowledge and join the discussion on shaping the future […]
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The US National Institutes of Health (NIH) announces a new collaboratory research opportunity on Newborn Screening by Whole Genome Sequencing. Eligible organisations are invited to submit their application before Friday, April 4th, 2025, local time of applicant organisation. LOIs are required to submit a full application. With significant community involvement and input, this initiative will […]
Read MoreIRDiRC is proud to have gathered experts, researchers, patient advocates, and industry leaders at its annual 2-days Consortium Assembly – Scientific Committees Meeting on 3-4 March, 2025, in Brussels, Belgium. Key takeaways from the meeting: These two days have been a testament to the importance of collective action. By breaking silos and fostering international cooperation, […]
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For the Rare Disease Day 2025, the European Commission has prepared a social media campaign on X and Facebook running from 10 to 25 February highlighting rare diseases projects ERICA and Solve-RD. In addition, a new updated factsheet will be available starting 28th February, together with a new Carousel-visual “4 facts you should know about […]
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A Day to Raise Awareness & Drive Action Rare diseases affect over 300 million people worldwide, yet many patients still face delayed diagnoses, limited treatment options, and a lack of awareness. Rare Disease Day is a global movement that unites researchers, advocates, policy makers, and patients to push for progress. In 2025, initiatives across multiple continents are bringing […]
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