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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IRDiRC has four Scientific Committees. The Therapies Scientific Committee (TSC) is a multi-stakeholder, multi-disciplinary group of experts in medical research and therapy development in rare diseases. Specifically, the TSC is devoted to pursuing the therapeutic development of IRDiRC, supporting the rare diseases research community in achieving goal 2, “1000 new rare disease treatments by 2027.” […]
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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 […]
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IRDiRC 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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The RealiseD project, funded under the Innovative Health Initiative (IHI), emerged in direct response to the strategic priorities set by the Rare Disease Moonshot. The Moonshot’s Clinical Trials Research Needs Recommendations provided a structured foundation that helped shaping RealiseD’s objectives, methodologies, direction and expected outcomes. The origins of RealiseD can indeed be traced back to the EFPIA-EURORDIS Joint Statement on […]
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We are pleased to share the webinar organized by the IRDiRC member, Rare Diseases International (RDI) – “From Grassroots to Global: Growing Momentum towards the WHA Resolution on Rare Diseases”. This year’s Rare Disease Day theme “More than You Can Imagine” reminds us of the milestones we have achieved by working together as a global community — […]
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Medicines Reimagined – The International Drug Repurposing Conference 2025 (#iDR25) hosted by REMEDi4ALL and Beacon aims to unlock the full potential of medicines by driving innovation for patient benefit. This global event will explore how cutting-edge artificial intelligence (AI) approaches are transforming drug discovery, alongside innovative business models and regulatory pathways for market access. It is a unique opportunity […]
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