The European Medicines Agency’s (EMA) Clinical Trials Information System (CTIS) has gone live as of January 31st. CTIS is the backbone of the Clinical Trials Regulation that will harmonise the assessment and supervision of clinical trials in the European Union. The Clinical Trials Regulation and CTIS will strengthen clinical trials in the EU, ensuring better…
The Therapies Scientific Committee of the International Rare Diseases Research Consortium (IRDiRC) is establishing a Task Force on Drug Repurposing Guidebook to help developers (of all kinds) navigating the rare disease landscape and identifying specific tools and practices of relevance for repurposing projects. The creation of the Development Guidebook will focus on repurposing approaches, following the same successful methodology used…
The Dravet Syndrome Foundation Spain works to improve the quality of life of people with Dravet syndrome and their families. The foundation recently released its end-of-year 2021 Dravet Syndrome Therapeutic Pipeline Report. In this report, the Foundation undertook a comprehensive review of the approved and investigational drugs and therapies for Dravet syndrome. The report also…
An EJP RD funded networking event on the rare neurological disease caused by anti-IgLON5 antibodies, discovered in 2014 by researchers from the Hospital Clínic-IDIBAPS is being organised in the context of the Networking Support Scheme (NSS) funding opportunity. The in person meeting will take place over two days on February 24th – 25th at the Esteve Auditorium of the Esther Koplowitz…
The International Rare Diseases Research Consortium (IRDiRC) is proud to announce the publication of a commentary on 10 years of progress and challenges of IRDiRC in the prestigious journal Nature Reviews Drug Discovery. The article summarizes IRDiRC’s vision and goals and highlights its achievements and prospects after its first decade (2011–2021). The commentary has been authored by Lucia Monaco…
Update: Third International Summit on Human Genome Editing postponed to 2023 The UK Royal Society, Academy of Medical Sciences, the US National Academies of Sciences and Medicine and UNESCO-The World Academy of Sciences for the advancement of science in developing countries (TWAS) are jointly organising the Third International Summit on Human Genome Editing in March…
The Therapies Scientific Committee of the International Rare Diseases Research Consortium (IRDiRC) is establishing a Task Force to characterize specific commonalities amongst a large group of “disregarded” rare diseases, with the potential secondary aims to identify removable roadblocks that may foster future research and development. The PLUTO project aims at using an integrated database search approach to: identify and classify…
The International Rare Diseases Research Consortium (IRDiRC) has published the results of its COVID-19 survey in the Rare Disease and Orphan Drugs Journal. The ambitious goals set by IRDiRC by 2027 to fulfill the vision of providing diagnosis and treatments to rare diseases (RDs) patients within one year of coming to medical attention have been challenged by the COVID-19…
The European Paediatric Translational Research Infrastructure (EPTRI) is organising a webinar tailored to the development of medical devices (MD), titled “A magnifying glass in the world of the medical device development”. The webinar will take place on January 20th, 2022 from 11.00 CET. This webinar concludes the series of webinars “A journey in paediatric medical devices: from regulations to clinical case studies and patients involvement”. During the…
The Innovative Health Initiative (IHI), a European Union public-private partnership funding health research and innovation, has published draft information on upcoming funding opportunities in 2022 in order to give potential applicants additional time to find or build a consortium and prepare a strong proposal. The first call for proposals will be launched in the first…