On September 16th 2019, a workshop “Rare disease perspectives in Central – Eastern Europe”, twinned to the General Assembly of EJP RD, will be organized in Gdansk, Poland. The main aims of this conference are discuss challenges and opportunities of rare disease research in Central – Eastern Europe countries and areas of involvement within EJP Read more about Rare disease perspectives in Central – Eastern Europe[…]
EMA’s human medicines committee (CHMP) has recommended the authorisation of Palynziq (pegvaliase), a new medicine for patients aged 16 and older with phenylketonuria, a rare but potentially serious inherited metabolic disease. Patients suffering from this disorder do not have the enzyme that breaks down phenylalanine, an amino acid which is found in most foods containing Read more about EMA’s human medicines committee (CHMP) recommends authorisation of new treatment for phenylketonuria, a rare inherited metabolic disease[…]
A new EURORDIS-led article, “Share and protect our health data: an evidence based approach to rare disease patients’ perspectives on data sharing and data protection – quantitative survey and recommendations” has just been published in the Orphanet Journal of Rare Diseases. The peer-reviewed article is freely accessed, it was written in collaboration with Rebecca Dimond, Read more about New results of #RareBarometer survey on patient’s preferences on rare disease data sharing & protection[…]
New Frontier Research – Health article by Horizon Magazine on a video at nanometre resolution -first time ever-, where scientists filmed how the immune system kills bacteria by poking holes into it. Professor Georg Fantner tells about how this was achieved and why his next challenge is to build a video camera that can film Read more about First ever nanoscale video on the immune system in action[…]
The success of European research missions, which aim to stimulate innovation to solve major societal problems, will require a complete change in the way governments think about business and risk, according to Professor Mariana Mazzucato, special advisor to the European Commissioner for Research, Science and Innovation, Carlos Moedas. On 4 July 2019, Prof. Mazzucato published a report Read more about FROM HORIZON 2020 TO HORIZON EUROPE: MISSION-ORIENTED APPROACH TO MAXIMIZE RESEARCH AND INNOVATION PROGRAMS IN EUROPE.[…]
A call for application for the preparation of EPTRI feasibility studies has been launched.Feasibility studies are aimed to test the process, the scientific interest, the availability of requested services and facilities needed to conduct paediatric research in the areas of EPTRI competence. Check it out!
Patients Active in Research and Dialogues for an Improved Generation of Medicines WP1 – Defining stakeholders’ preferences, needs and expectations This report describes the work that was done as part of PARADIGM’s work package 1 (WP1), which aimed to identify and define stakeholders’ preferences, needs and expectations in patient engagement (PE) through an online survey Read more about D1.2 Criteria to assess how patient engagement expectations are met[…]