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 […]
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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 […]
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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 […]
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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, […]
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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: […]
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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 […]
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 […]
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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 […]
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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 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 […]
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