News

European Union (EU) Health Summit – Rare Diseases Moonshot launch

Join us on 8th December for the European Health Summit that will take place in Brussels (Belgium) and online to find out more about our journey towards a Rare Disease Moonshot. Online registration is open here: https://lnkd.in/eCGr3ZmX IRDiRC Scientific Secretariat Coordinator, Daria Julkowska, will be part of the panel “Building a sustainable R&D ecosystem for […]

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IRDiRC Interview Series: Marjon Pasmooij

In the context of the IRDiRC Interview Series, Marjon Pasmooij’s interview is now available.  Marjon is a member of IRDiRC’s Therapies Scientific Committee and Science Programme Manager at Medicines Evaluation Board (CBG/MEB), University Medical Center Groningen, The Netherlands. Watch the full interview here

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Join IRDiRC’s Therapies Scientific Committee!

IRDiRC has four Scientific Committees, one each for Diagnostics, Therapies, Interdisciplinary, and Regulatory aspects of rare diseases research. The Therapies Scientific Committee (TSC) is a multi-stakeholder, multi-disciplinary group of experts in medical research and therapy development in rare diseases. Specifically, the TSC is devoted to pursuing the therapeutic development of IRDiRC, supporting the rare diseases […]

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FDA Rare Neurodegenerative Disease Grant Program

In addition to the Orphan Products Grants Program that the Office of Orphan Products Development (OOPD) currently administers, a new grant program was established this year by the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (ACT for ALS) named the FDA Rare Neurodegenerative Disease Grant Program. This new program will be administered […]

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IRDiRC Interview Series: Stefano Benvenuti

In the context of the IRDiRC Interview Series, Funders Constituent Committee member and Fondazione Telethon’s Head of Affairs Stefano Benvenuti’s interview is now available. “IRDiRC is a great opportunity for knowing more and doing your job better” says Stefano Benvenuti. Watch the full interview here

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Grant Funding Opportunity for Rare Diseases Research – Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases

The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds to support clinical trials for rare diseases and conditions. The purpose of this funding opportunity announcement is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in […]

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Science/AAAS Webinar: “Knowledge is power: The urgent need to internationalize databases for rare disease patients”

The recording of the Science/AAAS webinar on the subject “Knowledge is power: The urgent need to internationalize databases for rare disease patients” will be made available on September 29th at 12:00 PM EST. The webinar is expected to last approximately 60 minutes, during which a board of global experts (among which Lucia Monaco, former IRDiRC […]

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Fondazione Telethon participates in European AGORA Consortium

IRDiRC member organization Fondazione Telethon is delighted to share the press release of their participation in the European AGORA (Access to Gene therapies fOr RAre disease) Consortium. The AGORA Consortium was founded on September, 23rd 2022 with a focus on fostering alternative solutions to make life-saving gene therapies available for children born with rare genetic diseases. According […]

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