Please note: the hybrid event is open in person only to invited people. The event is open for all online.

Event Highlights:

• Dates: Monday, May 27 – Tuesday, May 28, 2024
• Venue: Hotel Excelsior Bari, Via G. Petroni, 15, Bari / Puglia, Italy
• Registration deadline: March 31st, 2024 – Registration is open here.

Agenda Coming Soon: Stay tuned as we prepare to unveil the detailed agenda, promising a comprehensive exploration of the impact of EJP RD.

More information on the event here.

Rare Disease Moonshot’s mission: Scaling up public-private partnerships to accelerate research by enhancing the translational research ecosystem, optimising clinical trials and regulatory pathways and develop infrastructure to accelerate the journey to diagnosis and treatment.

The paper is available here.

This open-access publication, reflecting the collaborative efforts of the IRDiRC Drug Repurposing Task Force and the IRDiRC Therapies Scientific Committee over the past 1.5 years, is a significant milestone in advancing drug repurposing for rare diseases.

The paper delves into key insights gained from analyzing cases of drug repurposing in small population conditions. Lessons learned include the importance of sharing clinical observations, early engagement with regulatory scientific advice, and fostering public-private collaboration.

The European Joint Programme on Rare Diseases is organizing the training webinar: “Real-World data, Machine learning and Deep analytics in rare diseases: Regulatory grade data collection for marketing authorization submissions – what is buzz, what is realistic?” that will be held online on 26 January 2024 from 2:00pm to 4:00pm CET.

Registration is open here before 25 January 2024 at 23:45 p.m

Marc Van Dijk (UCB) and Luis Pinheiro (EMA) will present views of industry and regulators on various facets of collecting, processing, and using data for research, regulatory and healthcare decisions. The two keynote talks will provide an overview of patient needs and challenges in rare disease R&D and of data and technologies that offer solutions to those challenges. 

The panel discussion between representatives of patients, academia, regulatory scientists, industry and clinicians and with the audience will provide different perspectives on challenges and solutions in the context of regulatory realities and imperatives. They will also provide examples of solutions already used and their limitations.

More information here.

Virginie brings an extensive professional background in rare diseases, scientific research, and patient advocacy, combined with personal insights from her niece’s experience with Congenital Disorders of Glycosylation.

EJP RD is proud to have collaborated with her at EJP RD, and excited to see her drive positive change in the rare disease community!

“I am privileged to re-join EURORDIS as CEO. My goal will be to build on both my professional experience and personal insights in rare disease research and patient advocacy to further EURORDIS’s mission, ensuring that the voices of all people living with a rare disease in Europe are heard and their needs are met,” Virginie stated.

Yann Le Cam, outgoing Chief Executive Officer of EURORDIS, expressed his full confidence in Bros-Facer’s leadership:

“I am thrilled to pass the baton to Virginie. Her unique blend of professional expertise and personal commitment to rare diseases makes her the ideal leader to guide EURORDIS into the future.”

Source: EURORDIS

Recordings of the event are available here!

Registration is open until 14 January 2024. Registration will remain open for the reserve list only until 4 February 2024.

Through the presentation of sample use cases that have long eluded diagnosis, the course will provide participants with with useful tools, instruments and knowledge on novel strategies to foster solutions of undiagnosed rare diseases cases. Moreover, the course will facilitate networking among professionals involved in undiagnosed rare conditions.

The course is open to the international research community, to clinicians and to medical specialists who have experience and concrete interest in the diagnosis and research on rare diseases.

To ensure active participation and exchange with teaching staff and participants, a maximum of 30 attendees will be admitted.

More information and registration here

This event has received funding support from the EJP RD’s Networking Support Scheme (NSS) funding opportunity.

A report has been prepared to understand more about the Argonaute syndromes, the event itself, and the feedback.

Read the report here.

ECRD is the largest, patient-led, rare disease policy-shaping event held in Europe, aimed at identifying key priorities to forge a unified approach for the next EU legislative cycle.

Registration will open in December

More information on what to expect here.