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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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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More than 90 healthcare professionals, leading experts and industry pioneers gathered for the 5th ICPerMed workshop on 14th and 15th November in Siena, Italy, to discuss latest technology developments and the frontiers of personalised medicine innovation. The prestigious one and a half day event at the Museum Santa Maria della Scala was organised by the […]
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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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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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The 12th European Conference on Rare Diseases and Orphan Products (ECRD) will take place on 15-16 May 2024, both online and in Brussels! It will be held as an official event under the auspices of the Belgian EU Council Presidency. ECRD is the largest, patient-led, rare disease policy-shaping event held in Europe, aimed at identifying key priorities to forge a unified approach for the […]
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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 […]
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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 […]
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The US National Institutes of Health (NIH) published a new Notice of Funding Opportunity (NOFO) with the aim to provide support for applications for IND-enabling studies for the development of a novel in vivo genome editing therapeutic platform (genome editor plus delivery system) for two or more disease indications, using the same genome editor, route of administration, and […]
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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 […]
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