The U.S. Food and Drug Administration (FDA) announces a new funding opportunity on “Natural History, Clinical Outcome Assessment, and Biomarker Studies of Rare Neurodegenerative Diseases (U01) Clinical Trials Optional”. The purpose of this funding opportunity announcement (FOA) is to support efficient natural history studies alone or in conjunction with the development and validation of clinical […]
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IRDiRC Functional Analysis Task Force recently convened for a dynamic 1.5-day workshop in Paris!🇫🇷 Members of the task force from all over the world came together and discussed methodologies and strategies to advance functional analysis for rare diseases. With collaborative spirit at the forefront, the task force members worked on putting together a manuscript on […]
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IRDiRC’s Interdisciplinary Scientific Committee (ISC) is looking for new members to join forces for exciting projects! Four positions will be available starting May, and the committee is looking for experts with different backgrounds, ideally based in Middle East, Africa, North or South America: Phenotyping, Ontology and/or Digital Health expertise Patient Advocacy experience Policy and/or Legal […]
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The Institute for Interdisciplinary Innovation in healthcare is organizing a symposium on “Gene therapy coming of age”, taking place on 28 March, 2024, at the Palace of Academies in Brussels, Belgium. The symposium will include sessions on: Recent research and clinical advances Patient perspectives Panel debate on economic and societal challenges I3H Annual Lecture “Gene […]
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As part of our commitment in advancing initiatives related to rare diseases, IRDiRC is a proud partner at the 2024 World Orphan Drug Congress USA. This will be a defining event for the rare disease and orphan drug space globally, bringing together top key opinion leaders from around the globe. The upcoming event will cover […]
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The International Niemann-Pick Disease Registry (INPDR) announced a new collaboration with Sanofi, by giving controlled access to anonymized registry information via INPDR’s research “Gateway” platform. This collaboration will provide Sanofi with Real-World Evidence that will support evaluation and decision making on the use of olipudase alfa to manage Acid Sphingomyelinase Deficiency (ASMD). INPDR is the […]
Read MoreWe are excited to announce that IRDiRC is now officially on LinkedIn! Get connected at: https://www.linkedin.com/company/international-rare-diseases-research-consortium-irdirc/about/
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Join the Canadian Organization for Rare Disorders (CORD) in celebrating the most impactful Rare Disease Day! This year’s edition marks the Anniversary of the announcement of Canada’s first National Rare Disease strategy with an allocation of 1.5 billion dollars over 3 years. In addition, CORD will celebrate the launch of the first ever Rare Disease […]
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Rare Disease Day is celebrated on the last day of February every year and is a special day for rare disease patients, researchers, and advocates. There are over 300 million people worldwide living with a rare disease, and together across borders and the 10,000+ rare diseases, we work towards more equitable access to diagnosis, treatment, care, […]
Read MoreIRDiRC is delighted to co-host, together with Hope for Rare Foundation and Fudan University, the Global Rare Diseases Research Symposium & The Second China Rare Disease Research and Translational Medicine Annual Conference on May 23-25 in Shanghai (China). The conference will last for 2.5 days with 21 parallel sessions and several satellite meetings. It will […]
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