The General Assembly of TEDDY Network will be held online, within a hearing at the Council of Europe this July 7th from 9.30 to 14.00 CEST. TEDDY Network will present some activities (part of which were developed within EJPRD) to be considered as good practices promoting the empowerment of children and facilitating their participation in…
EJP RD is delighted to announce the launch on June 30th, 2022 of the Innovation Management Toolbox (IMT), a free-to-use and curated reference library of resources in rare disease translational medicine that will provide investigators with self-help resources specific to their needs. The database will be maintained actively! Learn about the IMT: https://www.ejprarediseases.org/innovation-management-toolbox/ Access the…
The webinar on « Composite endpoints including patient relevant endpoints (Quality of Life) » is now available here In this webinar you will learn about: The procedures to combine multiple endpoints and its limitations The properties and the flexibility of the class of generalized pairwise comparison tests The potential advantages and disadvantages of designing a clinical trial…
Are you an adolescent aged from 12 to 18 interested in the themes of health, biomedical research, healthcare, and children rights?Are you a patient with a chronic rare disease interested in getting the skills to contribute in developing and shaping research making it more suitable for children?If yes, this paediatric expert patients training workshop is…
New opportunity to follow the MOOC on Rare Diseases Diagnostics! Given the enthusiasm raised by the MOOC « Diagnosing Rare Diseases: from the Clinic to Research and back » co-developed by Foundation For Rare Diseases, ERN Ithaca and ERN Genturis in the context of European Joint Programme on Rare Diseases, we have decided to keep it open continuously for enrollment.…
We are happy to invite you to take part in a workshop addressing “Ethics and regulatory considerations for ERN Data Access Committee members”.This workshop is jointly organised by ERICA and EJP RD and will take place online on 30th June from 10 am to 12:30 pm.It primarily aims to train the members of the ERNs Data Access Committee members…
Diamond-Blackfan anemia is a rare disease characterized by a defect in the production of red blood cells. In a plenary paper published in the journal Blood on May 26th, scientists highlighted a new form of the disease linked to the loss of function of the HEATR3 gene , the product of which chaperones the synthesis…
A funded networking event « European meeting on Phelan-McDermid syndrome » is being organised to discuss the guideline recommendations and to strengthen the collaboration that was started in 2020 by the European consortium on Phelan-McDermid syndrome (PMS) and supported by ERN-ITHACA, in order to tackle the knowledge gaps that they identified, as well as to discuss how best to…
A funded networking event « 2nd International Congress on Biliary Atresia and Related Diseases (BARD) » is being organised to offer access to the latest research and analysis related to this family of rare diseases. This event has received funding support from the EJP RD’s Networking Support Scheme (NSS) funding opportunity as part of the second round of funding…
A funded networking event “3rd International Symposium for Researchers and Clinicians on Wiskott Aldrich Syndrome (WAS2022)” is being organised to offer access to the latest research and analysis related to this rare disease. This event has received funding support from the EJP RD’s Networking Support Scheme (NSS) funding opportunity as part of the first round of funding…