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 […]
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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 […]
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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, […]
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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. […]
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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 […]
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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 […]
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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 […]
Read MoreWe are excited to announce the launch of several newly approved Task Forces and Working Groups focused on advancing the field of rare diseases. These groups bring together leading experts […]
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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 […]
Read MoreThe call for members for one Working Group and four new Task Forces is ongoing! The activities planned for 2025 are: (1) Task Force on “Preventive Medicines”: https://lnkd.in/eb4p-NSJPlease add in […]
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