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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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