News

“Drug Repurposing Guidebook” Task Force representatives will join the REMEDI4ALL iDR24 Conference

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 […]

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Happy Holidays from IRDiRC

IRDiRC 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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Just two months until application submission – FDA Office of Orphan Products Development Natural History Studies grant funding opportunity

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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CDER: Patient Community Engagement Public Meeting

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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The Chan Zuckerberg Initiative (CZI) is expanding the Rare As One Network and a new cycle will be open next year (including for organisations outside the USA). A call for applications is now in effect. CZI has funded 2 Cycles so far, and learned how successfully patient organisations are engaging in research collaborations, generating significant […]

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Scientific Call for Proposals AFM-TÉLÉTHON 2024

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 […]

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FDA Office of Orphan Products Development: Natural History Studies grant funding opportunity

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 […]

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The 2024 edition of the International Myotonic Dystrophy Consortium Meeting (IDMC-14) will be held in Nijmegen from 9-13 April 2024

Nijmegen is the oldest city in the Netherlands and one of the places where the disease-causing repeat expansion was identified in the nineties. OPTIMISTIC, the largest myotonic dystrophy trial, was initiated and coordinated in Nijmegen.   The meeting will offer an excellent opportunity to learn, share, and network with all those currently active in the field of myotonic dystrophy: […]

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