The video of Alex Sverdlov’s course on « Early Phase Trial designs in Rare diseases » is now live! Dive into the statistical methods crucial for developing novel therapies amidst challenges like small patient populations and ethical constraints. Watch the course here.
Watch the insights from the latest EJP RD interview with Sarah Weckhuysen, a researcher funded under EJP RD’s Joint Transnational Call. The collaborative approach of Sarah’s consortium, uniting experts across Europe which involves clinicians, scientists, and patient organizations, exemplifies a successful synergy for impactful research. Kudos to the team’s dedication and promising results! Watch her interviewMore…
Watch the insightful interview with Veronica Popa, a patient expert, as she delves into the subject of patient engagement in pre-clinical therapy development, the challenges, and the impact of EJP RD funding.The video is available hereIn this interview, she explores examples and discusses the challenges hindering patient participation in research projects.More information on EJP RD…
We are thrilled to share with you the new EJP RD video produced for ASHG TV which focuses on the value chain: from diagnosis to development of therapies for Rare Diseases. Together, EJP RD Members Dr. Daria Julkowska, Dr. Sonja van Weely, Dr. Ralph Schuster, Anton Ussi, Prof. Franz Schaefer, Dr. Ana Rath, Prof. Rima…
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…