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 […]
Read MoreIRDiRC 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 […]
Read MoreIRDiRC’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 […]
Read MoreThe Rare Disease Moonshot coalition encourages the rare disease research community to seize the opportunity presented by the Innovative Health Initiative’s call for proposal on Biomarkers, in alignment with the Research Needs Recommendations on Diagnosis In line with its mission of facilitating public private collaborations to address rare disease research “white spots”, the members of […]
Read MoreIRDiRC has just announced the launch of the latest publication titled “Advancing Diagnosis and Research for Rare Genetic Diseases in Indigenous Peoples” now available on nature.com.Read the publication here: https://www.nature.com/articles/s41588-023-01642-1“The Indigenous populations taskforce brought together Indigenous and non-Indigenous rare disease leaders from across the globe to tackle the inequity in rare diseases diagnosis. The outputs […]
Read MoreThe Interdisciplinary Scientific Committee (ISC) would like to broaden the committee membership. As such, the ISC has four openings for new members, specifically encouraging participants from the following regions: Middle East, Africa, North & South America. Applications from other areas will also be considered depending on the expertise. We are looking for rare disease experts with different backgrounds: Phenotyping, Ontology […]
Read MoreThe 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 […]
Read MoreJoin us in spreading awareness this Rare Disease Day.The International Rare Diseases Research Consortium (IRDiRC) is bringing together the efforts of member organizations. Check out this collaborative post showcasing their impactful contributions towards rare disease research this Rare Disease Day. Alexion AstraZeneca Rare Disease This year, to celebrate the Rare Disease Day, Alexion is hosting […]
Read MoreAs 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 […]
Read MoreThe 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 […]
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