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 […]
Read More
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 — […]
Read More
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 […]
Read More
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 MoreHosted 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 […]
Read More
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. […]
Read More
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 […]
Read More
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 […]
Read More
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 […]
Read More