AFM-Téléthon opens the call for proposals for 2024, open to both French and foreign teams, that aims to support research along the following thematics: A. Fundamental research and physiopathology of diseases of the neuromuscular system B. Development of innovative therapeutic approaches for rare genetic diseases The selected projects will be subject to an agreement with […]
Read More
The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) announces the 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 and standardized approaches to […]
Read More
The Rare Care Centre provides globally connected holistic cross-sector care coordination, together with other streams of activities to build capability and capacity to improve the lives of children and families living with rare diseases. The Centre provides a state-wide service based at Perth Children’s Hospital. For families living remotely, having a child with a rare […]
Read More
The Rare Disease Clinical Outcome Assessment (COA) is an initiative of the Critical Path Institute‘s Consortium with the same name, a public-private partnership that is focused on optimizing COA selection during the drug development for rare diseases. The COAs included in the the Rare Disease COA Resource represent a series of tools that are most […]
Read More
The Changing Focus of Regulatory Frameworks Around the Globe and the Opportunities for Harmonization
IRDiRC is proud to share that Regulatory Scientific Committee Vice Chair, Violeta Stoyanova-Beninska, is one of the guest editors of the research topic “The Changing Focus of Regulatory Frameworks Around the Globe and the Opportunities for Harmonization” in Frontiers in Medicine. As regulation related to the development, registration and monitoring of medicinal products is at […]
Read More
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 […]
Read More
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 […]
Read More
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 […]
Read More
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 […]
Read More
Under the Horizon Europe Framework Programme, a new two-stage call for proposals for “Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases” was published on 27 July, 2023. The project generated from the topic should not only develop capacities and capabilities to execute innovative trial designs, but also plan to identify solutions […]
Read More