The Rare Disease Cures Accelerator–Data and Analytics Platform is launching a new webinar series with the goal of sharing with the community examples of the use of rare disease person-level data in drug development and regulatory decision making. The series will feature 1-hour webinars highlighting analyses that have been done in individual disease areas, how…
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The 1st International Conference on Rare Diseases was held on the 1st and 2nd of March online. The event was a great success and was attended by more than 650 participants from 37 countries around the world (Europe, USA and Asia) including health professionals, researchers, academics, policy makers, industry representatives and patients. For the first…
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VISION-DMD published a white paper on how to return individual clinical trial results back to patients. It focuses on addressing the ethical and technical challenges, using the vamorolone clinical trial experience. In short: Often, individual patient data is not returned to the participants, even when this is requested. ReveraGen received a grant to develop an…
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We are glad to pre-announce the upcoming opening of the call for Research Mobility Fellowships on the 15th of March, which aims to support PhD students, Postdocs and medical doctors in training to undertake scientific visits fostering specialist research training outside their countries of residence. The exchange can be carried out within the same ERN…
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EMA has recommended granting a marketing authorisation in the European Union for the first treatment that can be given orally to patients with certain types of spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. Evrysdi is indicated for the treatment of 5q SMA…
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Happy Rare Disease Day! Watch EJP RD contributors answer the web’s most search questions about rare diseases in a new video that you can find HERE (subtitles available). Like every year, EJP RD is proud to contribute to spreading awareness on Rare Disease Day. Don’t forget to share the video and subscribe to our Youtube channel and to our Twitter to be kept updated on our funding opportunities,…
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(Pre-proposal deadline: 31 March 2021) Angelini Pharma is an international group, leader in health care and well-being, committed to help patients, doctors and caregivers in the fight against diseases with constant and prevalent attention in the areas of Pain and Inflammation and Mental Health. Angelini Pharma has made research its core business to improve patients’…
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The virtual event Unlocking innovation and access for rare disease patients in Europe was held online on the 22nd of February. With over 700 registrations from across the European rare disease community, the event was full of insights on the impact that policy decisions have the quality of lives of people affected by rare diseases.…
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Heritable human genome editing has been proposed as a means of helping parents avoid passing genetic diseases to future generations. But can heritable human genome editing be used safely? On February 26, 2021 at 9:00 am EST (2 pm GMT / 3pm CET / 10 pm CST), join members of the International Commission on the…
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The International Rare Diseases Research Consortium (IRDiRC) is glad to announce the opening of two new Task Forces: “Shared Molecular Etiologies Underlying Multiple Rare Diseases” aiming to address and document the existing challenges in adapting the basket trial approach used in molecularly targeted oncology clinical trials to drugs targeting shared molecular etiologies underlying multiple rare…
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