Category: IRDiRC

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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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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