The Joint Action on Integration of ERNs into National Healthcare Systems (JARDIN) project has put out their first Bullet Point Newsletter. JARDIN is a huge and complex project that is met with very high expectations from EC and members. Check it out!
The Cross-Border Access to Paediatric Clinical Trials Working Group (WG) created by the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) is collecting data. The aim of this WG is to facilitate the inclusion of children and young people in clinical trials across Europe avoiding any language barriers. Recently we have identified that some patients could not take part in a clinical trial because they do not speak the official language of the country where the trial is performed or English.
Enpr-EMA is currently collecting data from Clinical Research Units and, also from parents of children living [...]
EURORDIS recently published the results of their full Rare Barometer survey which can be found here. The key findings are available in multiple languages.
Key findings include:
4.7 years for a confirmed diagnosis 40% have NOT been referred to a Centre of Expertise 25% had 8 consultations or more with healthcare professionals before their diagnosis was confirmedEURORDIS also provided a breakdown of the responses related to rare endocrine conditions.
Four European Reference Networks (ERN-EYE, ERKNet, Endo-ERN, ERN-ITHACA) have teamed up to establish a consensus statement and recommendations for Bardet-Biedl syndrome (BBS) providing guidance for the relevant experts.
“This consensus statement also emphasizes on initial diagnosis, monitoring and lifelong follow-up, and symptomatic care that can be provided to patients and family members according to the involved care professionals.”
Endo-ERN ePAG Bernd Rosenbichler shared a recent event organised by a local representative that featured his talented son Ben.
“Erich Irlstorfer, member of the German Bundestag, took up the topic of rare diseases almost 1.5 years ago – as a non-partisan initiative. He organized over 60 events with several thousand participants on the topic of Rare in Bavaria. He is in talks with European politicians to give the topic even more space. Other German states are following and also taking action. He brings his experiences and demands to the German Bundestag and to the political committees.
On July 13, he [...]
The Joint Action on Integration of ERNs into National Healthcare Systems (JARDIN) project has put out their first Bullet Point Newsletter. JARDIN is a huge and complex project that is met with very high expectations from the EC and member states. Check it out!
The Endo-ERN covers under its umbrella a broad range of endocrine disorders, and a significant number of these entities are caused by genetic variants. The underlying disorders are commonly rare and research, diagnostic testing and treatment are fragmented. Clinically, the disorders covered by Endo-ERN are heterogeneous, as are their genetic basis.
However, the genetic mechanisms behind the disorders as well as the genetic testing strategies are very similar. The last years have seen considerable improvement of genetic testing and – as a consequence – of the development and implementation of personalized treatment strategies.
The need to implement and run [...]
July 2025 The survey is now closed and the research complete.
Walden University in Minneapolis, Minnesota, USA has a new study about the experiences of managing and living with autoimmune Addison’s disease in the younger and newly diagnosed adults. Researcher Mathew Horodner is seeking English speaking patients aged 18-30 for a video interview that will audio-recorded.
The criteria has been expanded and you may now qualify to particpate. Check out the new flyer or contact researcher Mathew Horodner.
The new CPMS 2.0 system went live 31 May, 2024.
For the next few months Endo-ERN will continue to use the original CPMS, as we start preparation for a network-wide migration to the new CPMS 2.0 system. Existing users will automatically have an account in the new system once data migration occurs, using their same login details for EU login. If you want to test the system ahead of our network’s formal migration, please contact our helpdesk.
With the new system comes formal steps for each of our member HCPs. The new system security documentation will be shared [...]
Exciting News! The EJP RD course “Innovative Therapies and Personalized Medicine for Rare Diseases” is officially live! Explore cutting-edge advancements in gene therapy, regenerative medicine, and beyond. Be a part of the revolution in rare disease treatment! Enroll now.
This course is a free course until Monday, 12 August.
