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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The World Duchenne Organization is pleased to announce that the WDO Leadership Award, the highest recognition for individual achievement, was awarded to Professor Francesco Muntoni on September 7, 2022. Prof Muntoni is Director of the Dubowitz Neuromuscular Centre at Great Ormond Street Institute of Child Health and Great Ormond Street hospital. This award acknowledges and celebrates…
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As a continuation of the funding of an EJP RD NSS call, ECRIN is holding a two-day workshop and networking event in Prague on October 4th & 5th; the event will see the gathering of investigators involved in drug repurposing academic-sponsored trials for rare diseases, as well as other rare diseases clinical trial stakeholders. To improve the existing treatments of…
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IRDiRC, a global collaborative initiative with the vision to enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention, today announced the formation of the Regulatory Science Committee (RSC). The new RSC “brings broad expertise across different stakeholders and geographies allowing…
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EJP RD held its annual General Assembly and Consortium Meeting on 13-15 September 2022 in Porto, Portugal. The event gathered over 100 participants from various institutions across Europe and associated countries. It was an opportunity to bring back together EJP RD members to meet in-person. Collaboration, Innovation, Data, Funding & Empowerment are key for the…
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EURORDIS is delighted to announce that applications for the 2023 edition of the EURORDIS Open Academy Schools are now open. The 2023 edition of the Open Academy Schools will take place in-person from 19-23 June in Barcelona and, following a review of our training offerings, we are excited to announce a new format for our…
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The 4th edition of the Neuromuscular Translational School organized by EURO-NMD and TREAT-NMD will take place in autumn this year from November 21-25 in Leiden University Medical Center, in the Netherlands. The Translational School focuses on therapy development from bench to bedside with a focus on neuromuscular disorders. It contains lectures about the current state…
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EJP RD is delighted to announce an upcoming webinar on « The Statistical Evaluation of Surrogate Endpoints in Clinical Trials » which will take place online, on November 18, 2022, at 16:00 CEST. The webinar will be divided in two parts; a first half of the webinar will be devoted to the in-depth review of surrogate marker…
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The French Foundation for Rare Diseases is delighted to announce the upcoming opening of a new facilitation window of the MOOC “Diagnosis Rare Diseases: from the clinic to research and back” on September 19th. This update will be available until December 3rd 2022. During this period, expert mentors will be actively present on the platform to answer the…
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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 updated by the…
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