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.…
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…
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…
On the occasion of Rare Disease Day 2021, Biogen is pleased to invite you to the virtual event: “Unlocking innovation and access for rare disease patients in Europe” taking place on Monday, 22 February from 14:00 to 15:45 CET. Biogen’s event, done in collaboration with Reuters and EU40, will highlight the need for continued political…
Ring14 International yearly solicits international research projects focused on the Ring 14 or chromosome 14 related syndromes. The projects can be basic or pre-clinical, the ultimate goal is to find a cure for these conditions. The Board of Directors of Ring 14 International, is pleased to inform that the 2021 call for applications has been issued…
The European Medicines Agency (EMA) has published for public consultation a draft toolbox guidance on scientific elements and regulatory tools to support quality data packages for PRIME marketing authorisation applications. The EMA launched the PRIME scheme to enhance support for the development of medicines that target an unmet medical need. This voluntary scheme is based…
The Food and Drug Administration will be holding a virtual public meeting on Friday, March 5, 2021, from 9:00 a.m. to 4:00 p.m. EST to highlight strategies to support rare disease product development. Please visit the public meeting page to register for the event. To learn more about other activities relevant to rare diseases and…
If you are interested in participating in an initiative in order to contribute to the implementation of the children rights in research and medical field, the Council of Europe launched an initiative on children rights participation in decision making in the biomedical field. The UN Convention on the Rights of the Child and other Human…
For the first time, EURORDIS, alongside its Council of National Alliances, Council of European Federations and its members, have set out 11 Key Principles to support a harmonised European approach to Newborn Screening. The vast inequalities across Europe, coupled with technological and scientific advances highlight the urgent need to move forward from the status quo. The…
The ERA-NET Neuron Call for proposals for Transnational Research Projects on Neurodevelipmental Disorders was launched January 7 and will close March 9 14:00 CET Neurological and mental disorders impose a high societal and economic burden to the European population, leading to costs of more than 800 billion euros per year. A wide range of brain…