IRDiRC is proud to announce the publication of the latest article in Therapeutic Advances in Rare Disease: “From Roadmap to a Sustainable End-to-End Individualized Therapy Pathway.” This publication builds on the foundational work of the IRDiRC N-of-1 Task Force, expanding from the initial roadmap to explore how N-of-1 therapies—custom treatments for individuals with ultra-rare genetic conditions—can […]
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IRDiRC has four Scientific Committees: Diagnostics, Therapies, Interdisciplinary, Regulatory. The Interdisciplinary Scientific Committee (ISC) is a multi-stakeholder, multi-disciplinary group of experts working on rare disease (RD) data sharing, ontologies, natural history, biobanks, registries, ethical and societal aspects of rare diseases. ISC would like to broaden the committee membership, to represent the worldwide rare diseases community in its […]
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On the 25th of April, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) gave givinostat (Duvyzat), a novel histone deacetylase (HDAC) inhibitor, a positive opinion. The recommendation is for granting a conditional marketing authorisation for givinostat for the treatment of ambulant patients with Duchenne Muscular Dystrophy (DMD) aged six […]
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Commissioned by the French Ministry of Higher Education and Research, ANR (Agence nationale de la recherche) will launch a specific call for research projects on Charcot disease, also known as Amyotrophic Lateral Sclerosis (ALS), a neurodegenerative disease caused by the progressive degeneration of the central and peripheral motor neurons in the brainstem and spinal cord. […]
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“Breaking down silos and fostering collaboration to help rare disease patients” – The World Orphan Drug Congress convenes top pharmaceutical and biotech companies, public and private funders, patient advocacy groups and many more. IRDiRC is delighted to confirm the participation of Dr. David Pearce at the WODC USA during the 3 congress days, in the […]
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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 […]
Read MoreWe 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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