Category: IRDiRC

CORD Rare Disease Day Summit

The Canadian Organization for Rare Disorders (CORD) is very excited to be celebrating this once-in-four- years February 29th Rare Disease Day in Ottawa (Canada) on February 28-29, 2024. A panel, “Canadian Pathway to Rare Disease Drug Access”, is also scheduled on February 28 at 10:45 AM – 12:00 PM (local Ottawa time). This year, there […]

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New IRDiRC Publication – Drug Repurposing for Rare: Progress and Opportunities for the Rare Disease Community

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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EJP RD is partnering with EFPIA for the Webinar: Real-World data, Machine learning and Deep analytics in rare diseases

The European Joint Programme on Rare Diseases, together with the Companies Constituent Committee (CCC) member EFPIA, is organizing the training webinar: “Real-World data, Machine learning and Deep analytics in rare diseases: Regulatory grade data collection for marketing authorization submissions – what is buzz, what is realistic?” that will be held online on 26 January 2024 […]

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Project CASK Grant Program – Now accepting applications!

Project CASK, in collaboration with Orphan Disease Center, is seeking proposals to advance research that supports therapeutic development for CASK gene disorders. The project will consider applications that will make a significant impact on the CASK community and the prospects for accelerating therapeutic development in one of two categories: 1) Grants to expand the study […]

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12th European Conference on Rare Diseases & Orphan Products 2024

The European Conference on Rare Diseases & Orphan products (ECRD) is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place. This 12th edition will […]

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2024 Biannual CUPA Conference: Hot Topics in Expanded Access

Join us for this 2-day multi-stakeholder event on 29-30 January covering the day’s most pressing ethical issues in non-trial preapproval access at CUPA Con 2024! In-person attendees will be able to take advantage of interactive workshops, various networking opportunities, a cocktail reception, and other perks. For those unable to attend in-person, tickets providing access to […]

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Apply now for the CDKL5 Pilot Grant Program

The Orphan Disease Center (ODC) at the University of Pennsylvania, in collaboration with the Loulou Foundation, is pleased to announce the 2024 CDKL5 Program of Excellence Pilot Grant Program. The deadline for the Letters of Interest (LOIs) is Friday, February 16, 2024, 5pm EST.      CDKL5 Deficiency Disorder (CDD) is a monogenic, neurodevelopmental disorder characterized […]

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“Drug Repurposing Guidebook” Task Force representatives will join the REMEDI4ALL iDR24 Conference

IRDiRC is delighted to announce that multiple representatives of the Task Force “Drug Repurposing Guidebook”, including Therapies Scientific Committee (TSC) vice chair Anneliene Jonker (Duchenne Parent Project, The Netherlands) and TSC member Marjon Pasmooij (Medicines Evaluation Board, The Netherlands), will present at the International Drug Repurposing Conference, #iDR24, co-organised by REMEDi4ALL, Beacon for Rare Diseases […]

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