The European Brain Council (EBC) is organizing a meeting at the European Parliament, in Brussels (Belgium), on 14 November 2023, 8:00-9:30 AM CET to discuss “Good off-label use practices: Where are we? What next?“. Marc Dooms, IRDiRC Interdisciplinary Scientific Committee Vice Chair, will offer a presentation about the off-label use of devices. The goals of this event are to reconnect […]
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Don’t miss out the last event of the Reuters Hemophilia Changemakers 2023 Series – in partnership with Sanofi – that consists of three virtual events, two that took place in June and September, and the last one planned for November 7, 2023. It will bring together esteemed thought leaders to discuss policies, practices, and outline […]
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The commentary ‘IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases’ has been published in Nature Reviews Drug Discovery (https://www.nature.com/articles/d41573-023-00168-9). This guidebook, authored by the IRDiRC Task Force “Drug Repurposing Guidebook”, facilitates drug repurposing for rare diseases by organizing available tools into a standardized framework. The Task Force worked for […]
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The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal year (FY) 2024 to support natural history studies for rare diseases and conditions. This funding opportunity is intended to support prospective or retrospective, efficient, and innovative natural history studies with a focus on collaborative […]
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The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal years (FY) 2023 – 2025 to support clinical trials for rare diseases and conditions. The purpose of this funding opportunity announcement is to fund clinical trials of products evaluating efficacy and/or safety in support of a new […]
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On October 14, 2023, the press conference of “Breaking the Last Mile – Research Report and Policy Recommendations on the Implementation of Rare Disease National Drugs (2023)” was successfully held by Chinese Organization for Rare Disorders (CORD) in Shanghai! Since the Chinese National Medical Insurance Administration – China Healthcare Security (CHS) launched the National Medical […]
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The IQSEC2 Research and Advocacy Foundation will provide a 1-year grant, with a no cost extension of up to one year granted with approval, to support research related to IQSEC2-related disorder. The foundation will award either one grant of $60,000 or two grants for $30,000. The Foundation, in collaboration with the Orphan Disease Center, is […]
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The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal years (FY) 2023 – FY 2025 to support clinical trials for rare diseases and conditions. The purpose of this funding opportunity announcement is to fund clinical trials of products evaluating efficacy and/or safety in support of a […]
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In the context of the Belgian Presidency of the Council of the European Union, the Belgian minister of health wants to make a focal point of identifying and using evidence on unmet patient and societal needs, with the objective of developing a healthcare system and policy that is more needs- than supply-driven. KCE and Sciensano […]
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