Project CASK, in collaboration with Orphan Disease Center, is seeking proposals to advance research that supports therapeutic development for CASK gene disorders. The project will consider applications that will make a significant impact on the CASK community and the prospects for accelerating therapeutic development in one of two categories: 1) Grants to expand the study […]
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The European Conference on Rare Diseases & Orphan products (ECRD) is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place. This 12th edition will […]
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The Innovative Health Initiative (IHI) has now launched IHI – call 6 and IHI – call 7. IHI – call 6 is a two-stage call for proposals with the following topics: Topic 1: Support healthcare system resilience through a focus on persistency in the treatment of chronic diseases Topic 2: Development of evidence based practical […]
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Join us for this 2-day multi-stakeholder event on 29-30 January covering the day’s most pressing ethical issues in non-trial preapproval access at CUPA Con 2024! In-person attendees will be able to take advantage of interactive workshops, various networking opportunities, a cocktail reception, and other perks. For those unable to attend in-person, tickets providing access to […]
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The Orphan Disease Center (ODC) at the University of Pennsylvania, in collaboration with the Loulou Foundation, is pleased to announce the 2024 CDKL5 Program of Excellence Pilot Grant Program. The deadline for the Letters of Interest (LOIs) is Friday, February 16, 2024, 5pm EST. CDKL5 Deficiency Disorder (CDD) is a monogenic, neurodevelopmental disorder characterized […]
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IRDiRC is delighted to announce that multiple representatives of the Task Force “Drug Repurposing Guidebook”, including Therapies Scientific Committee (TSC) vice chair Anneliene Jonker (Duchenne Parent Project, The Netherlands) and TSC member Marjon Pasmooij (Medicines Evaluation Board, The Netherlands), will present at the International Drug Repurposing Conference, #iDR24, co-organised by REMEDi4ALL, Beacon for Rare Diseases […]
Read MoreIRDiRC in 2023 – Four new Task Forces, numerous publications, RE(ACT) Congress, World Orphan Drug Congress participation and the list goes on! We couldn’t have had such a successful year without you! We are sending our sincere gratitude to the Rare Disease community for the fantastic work and involvement towards the common mission of improving […]
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The grant application submission deadline is coming up in just 2 months! The application submission deadline for this grant funding opportunity is February 13, 2024. Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01) The purpose of this grant is to support efficient and innovative natural history studies that advance medical product development in rare […]
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Have a look at the latest video from the IRDiRC Consortium Assembly Meeting hosted on 3-4 October in Montréal (Canada) and dive into insightful snippets from different committees discussions! IRDiRC Consortium Assembly members had a chance to provide live updates on the activities developed within their committees and offered an insight on the priorities for […]
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On December 14, CDER and Duke-Margolis will host a jointly sponsored virtual public meeting on advancing the development of therapeutics through rare disease patient community engagement. The purpose of this public meeting is to highlight and build upon existing actionable approaches for engaging patients, patient groups, and related experts when developing necessary evidence to support […]
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