A roundtable of EU and national policymakers, industry experts and patients will gather together to discuss the best approach to define unmet medical needs and Belgium’s multi-criteria approach. Around 36 million Europeans suffer from rare diseases and 95% of these diseases lack a specific treatment. Moreover, medicine development is lagging due to a risky and […]
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The latest IRDiRC paper, “Drug Repurposing for Rare: Progress and Opportunities for the Rare Disease Community” has been published in Frontiers in Medicine. The paper is available here. This open-access publication, reflecting the collaborative efforts of the IRDiRC Drug Repurposing Task Force and the IRDiRC Therapies Scientific Committee over the past 1.5 years, is a […]
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Have a look at the latest video from the IRDiRC Consortium Assembly Meeting hosted on 3-4 October in Montréal (Canada) and dive into insightful snippets from different committees discussions! IRDiRC Consortium Assembly members had a chance to provide live updates on the activities developed within their committees and offered an insight on the priorities for […]
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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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IRDiRC proudly announces the publication of the manuscript “Targeting shared molecular etiologies to accelerate drug development for rare diseases“ online, today. The manuscript is published in the EMBO Molecular Medicine Journal and freely accessible online at the following link: https://www.embopress.org/doi/full/10.15252/emmm.202217159.
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The International Rare Diseases Research Consortium (IRDiRC) announces new leadership changes in its Funders Constituent Committee (FCC). Dr. Adam Hartman (Program Director at the National Institute of Neurological Disorders and Stroke (NINDS), USA), FCC Chair, and Dr. Catherine Nguyen (Director at the Thematic Institute of Genetics, Genomics, and Bioinformatics, INSERM, France), FCC Vice Chair, have […]
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The Therapies Scientific Committee (TSC) would like to broaden the committee membership, to represent the worldwide rare disease therapies community in its full width, from early innovation towards regulatory approval and therapeutic support of patients. As such, the TSC has three openings for new members, specifically encouraging participants from the following regions: Africa, Asia, Australasia […]
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IRDiRC is thrilled to present a new video from the Interview Series with its members. This month we have in the spotlight David Adams, IRDiRC Diagnostics Scientific Committee (DSC) Chair and Deputy Director of Clinical Genomics at NHGRI/NIH (USA). Check out David’s answers on what IRDiRC means for him and the role it plays for […]
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Prof. Guillaume Canaud (Necker Hospital for Children, AP-HP, France) offered a great presentation on “Targeted therapy for patients with PIK3CA-related overgrowth spectrum“ at the RE(ACT) Congress in Berlin (Germany). Check out his impressions on the IRDiRC Conference x RE(ACT) Congress at the following link: here.
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The European Partnership on transforming health and care systems (THCS), a Cofund action under the Horizon Europe Programme designed to support coordinated national and regional research and innovation programmes along with capacity building, networking, dissemination and other key activities to support health and care systems transformation, will launch its first Joint Transnational Call (entitled “Healthcare […]
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