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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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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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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The International Rare Diseases Research Consortium (IRDiRC) and the European Joint Programme on Rare Diseases (EJP RD) are jointly organizing a two-day conference on clinical research networks (CRNs) for rare diseases, which will take place on December 1 and 2. The objective of this conference is to gather experts from different continents to increase mutual knowledge […]
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IRDiRC is delighted to share the release of Connecting the dots: Embedding progress on rare disease into healthcare, an Economist Impact report sponsored by Takeda, a company that is part of IRDiRC’s Companies Constituent Committee. The report, to which IRDiRC members Takeya Adachi, Gareth Baynam and Hugh Dawkins contribute as an expert panel, takes a […]
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IRDiRC Member Organization Rare Diseases International is delighted to announce their upcoming webinar on the use of the International Classification of Diseases 11th Revision (ICD -11) for rare diseases, which will take place on September 27th, at 14.00 CET. The webinar will be co-hosted by RDI and the World Health Organization. The ICD is developed and […]
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Global Genes is delighted to announce that they will soon be hosting their largest event of the year, the Patient Advocacy Summit. The 3-day event will take place from September 12th to September 14th in San Diego, California (USA). Join patients, caregivers, advocates, healthcare professionals, researchers, partners, and allies of the rare diseases community; hear […]
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EURORDIS is pleased to announce that nominations for the EURORDIS Black Pearl Awards are now open until September, 4th. The Awards are presented to patient advocates and organisations, policy makers, scientists, companies, and media in recognition of their exceptional achievements and outstanding commitment to the rare disease cause. NOMINATIONS: Just like every year, nominations can be submitted for any individual, organisation or company (including yourself and/or […]
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As one of the outcomes of the MedTech for Rare Diseases Working Group led by IRDiRC members Anneliene Jonker and Marc Dooms, the Orphanet Journal of Rare Diseases has decided to focus a thematic issue on the questions regarding the research and development of orphan devices and medical technologies that are being developed for Rare Diseases […]
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London, UK – July 2022: The RDNN Board of Trustees and Management is delighted to announce the recent registration of RDNN as a registered UK charity. This is an important milestone as they strive forward on their mission to provide a network of specialist nurse support for every person with a rare disease. RDNN has […]
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