The course will cover the path to follow for exploiting the high value of the ERNs (European Reference Networks) biological samples repositories in the rare diseases (RD) research.

Attendees will learn about the role of ERNs samples and how to optimize them for networking and research networks.
Don’t miss this opportunity to enhance your knowledge and skills in the field of rare diseases research!

More information here

The in-person event will take place on 8-9 May 2023 at Van der Valk Hotel in Nijmegen-Lent, The Netherlands.

Registration is open here, and closes on February 1st.

The workshop, open to all research levels and aims to to raise awareness for the psychosocial impact of having a rare disease and form a group of researchers in the mental well-being and social sciences domain for rare diseases. 

More information here

In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled “EJP RD – ERN Workshop: New Therapeutical Approaches for Inherited Retinal Dystrophies” is being organized by Dr Regina Rodrigo.

The in-person event will take place on 27-28 March 2023 at CIPF – Príncipe Felipe Research Center, Eduardo Primo Yúfera, 3, 46012, Valencia, Spain.

Registration is open here, and closes on December 31st.

The workshop, open for beginners and medium level researchers – junior physicians and scientists, will give an update on basic and translational research related to new therapeutic approaches for IRDs. It will bring together European experts and trainees to improve the training of young researchers on IRDs and latest therapies. Participants and speakers will involve representatives from ERN-EYE partners and non-ERN-EYE partners. 

More information here

In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled “Epidermolysis bullosa: from genes to translation into therapies” is being organized by Prof. Dr. Cristina Has of the University Hospital Freiburg.

The in-person event will take place on 23-24 March 2023 in CIBSS – Centre for Integrative Biological Signalling Studies in Freiburg, Germany.

Registration is open here, and closes on December 15^th.

The workshop, open for beginners and medium level researchers – junior physicians and scientists, will deliver expert knowledge on the following aspects of EB: clinical and genetic basis, wound and interdisciplinary management, how to design and conduct clinical trials, gene therapy, and models used in translational research. The aim is preparing the next generation of EB researchers.

More information here

Together, EJP RD Members Dr. Daria Julkowska, Dr. Sonja van Weely, Dr. Ralph Schuster, Anton Ussi, Prof. Franz Schaefer, Dr. Ana Rath, Prof. Rima Nabbout, Dr. Roseline Favresse, and Prof. Biruté Tumiene highlight different elements of the EJP RD in the broader context (including genetics and gene therapies), as well as future plans of the programme.

Watch the full video here

We are glad to announce the launch of the new MOOC entitled “Introduction to translational research for Rare Diseases” will be available.

On this five-week course from the European Joint Programme on Rare Diseases, you’ll explore the therapeutic development process from the unique perspective of rare diseases.

You will follow the journey from discovery to approved treatment, learn how therapeutic targets and drug candidates are identified and validated, and examine the special regulatory frameworks.

This course is designed for researchers and students in medicine and health-related research fields, as well as healthcare professionals wanting to further their knowledge of translational research in rare diseases.

It is also suitable for biotech and start-up developers and Patients Advocacy Organisation representatives who want to increase their understanding of current practices in therapeutic developments for rare diseases.

More information here

On October 17th, a new MOOC entitled “Introduction to translational research for Rare Diseases” will be available.

On this five-week course from the European Joint Programme on Rare Diseases, you’ll explore the therapeutic development process from the unique perspective of rare diseases.

You will follow the journey from discovery to approved treatment, learn how therapeutic targets and drug candidates are identified and validated, and examine the special regulatory frameworks.

This course is designed for researchers and students in medicine and health-related research fields, as well as healthcare professionals wanting to further their knowledge of translational research in rare diseases.

It is also suitable for biotech and start-up developers and Patients Advocacy Organisation representatives who want to increase their understanding of current practices in therapeutic developments for rare diseases.

More information here

Read the article here (open access)

Related article: ECRIN Networking Meeting Identifying obstacles hindering the development of academic-sponsored trials for drug repurposing on rare-diseases

The International Rare Diseases Research Consortium (IRDiRC) and the European Joint Programme on Rare Diseases (EJP RD) are jointly organizing a two-day conference on clinical research networks (CRNs) for rare diseases, which will take place on December 1 and 2. The objective of this conference is to gather experts from different continents to increase mutual knowledge on CRNs structure, activities and identify pathways to stimulate collaboration and interoperability of these networks.  

It is not possible anymore to register for in-person participation. To register for online participation, please complete this registration form. 

Find more information here.

EJP RD held its annual General Assembly and Consortium Meeting on 13-15 September 2022 in Porto, Portugal. The event gathered over 100 participants from various institutions across Europe and associated countries. It was an opportunity to bring back together EJP RD members to meet in-person.

Collaboration, Innovation, Data, Funding & Empowerment are key for the European Joint Programme on Rare Diseases to attain its goal of creating an effective rare diseases research ecosystem for progress, innovation and for the benefit of everyone with a rare disease.