A funded networking event “Treat AIE: First European networking conference to provide guidelines and improve treatment of Autoimmune Encephalitis” is being organised to foster collaboration and improve the future of Autoimmune Encephalitis (AIE). This event has received funding support from the EJP RD’s Networking Support Scheme (NSS) funding opportunity as part of the third round of funding…
The third run of the MOOC (Massive Open Online Course) “Diagnosing Rare Diseases: from the Clinic to Research and back” co-developed by the European Joint Programme on Rare Diseases, ERN Ithaca, ERN Genturis and the French Foundation for Rare Diseases starts TODAY (April 18th)! Registrations are free and open at this link. We specifically encourage medical and biomedical science students to register…
The Share4Rare Consortium is organising a webinar to discuss the potential of clinical imaging in rare disease diagnosis and research. The guest speaker will be Dr. Josep Munuera, radiologist and Head of the Quality, Innovation and Research Section of the Diagnostic Imaging Service at Hospital Sant Joan de Déu Barcelona, Spain. The webinar will take place on April 20th from 17.00 – 18.00 CET.…
A recent report of the joint action Towards the European Health Data Space (TEHDAS) examines EU-level and national funding for health data collection, access and use for secondary purposes, such as research. To ensure the economic sustainability of the forthcoming European health data space (EHDS), the EU and the member states will need to solve how…
A recent report of the joint action Towards the European Health Data Space (TEHDAS) identifies a minimum set of technical services – basic software items – required for the cross-border secondary use of health data in Europe. The secondary use of health data involves using health data, such as patient records, for research, decision-making, and…
The National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS) and the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) are hosting a jointly sponsored virtual workshop to explore Regulatory Fitness in Rare Disease Clinical Trials. The virtual workshop will take place over two days on May 16th – 17th. Academic investigators, patient…
The European Medicines Agency (EMA) is organising a joint EMA/HMA/TEHDAS Data Quality Framework for Medicines workshop as a virtual meeting on April 7th from 13.00 – 17.15 CET. This workshop seeks to engage stakeholders early in the drafting process of an EU Network data quality framework, to ensure a robust starting point, in line with their…
As part of the EJP RD Training series, an advanced-level training webinar on the topic « Composite endpoints including patient relevant endpoints (Quality of Life) » aimed at introducing the generalized pairwise comparison ideas and concepts as well as demonstrating its benefit in small sample trials, more particularly in including patient relevant endpoints, such as quality of…
In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled « Modelling & Simulation: Research Methodologies for Small Populations in Rare Diseases » aimed at facilitating discussion and exchange of knowledge on the M&S methodologies and strategies as innovative and promising enough for facing complex multifactorial or rare diseases and conditions that require highly specialised treatments and resources is being organised…
In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled “Functional exploration of genetic variants in cardiac diseases” aimed at presentation of functional explorations of variants of interest identified using Drosophila, C. elegans, Zebrafish and iPSc models to understand the molecular bases of genetic diseases, in particular heart and muscle diseases, is being organised by Philippe Chevalier of the Hospices Civils de…