Endo-ERN ePAG, Nathalie Ferard represents Association GRANDIR and has kindly provided an update newsletter on the many activities and resources available from the organisation.
Activities/resources include:
participation at national and international medical meetings family videoconference meetings to discuss growth hormone issues podcast on injection ritual to help reduce potential anxiety (French) supporting Miss Small Beauty competition
To mark Rare Disease Day, the European Commission published several resources celebrating the dedication and expertise of the European Reference Networks that make a tangible difference to the lives of patients with rare diseases and their families. Check them out below!
EU delivering on rare diseases for patients and families
How the European Commission works for Rare Diseases and the European Reference Networks (ERNs)
European Reference Networks: A success story for patients living with rare disease
New evidence-based guidelines were recently published. Topics covered include:
Diagnosis of XLH Initial evaluation of the presence and severity of complications of |X-linked hypophosphaeaemia Definitions of responses to therapy in patients with X-linked hypophosphataemia Recommendations for follow-up of patients with X-linked hypophosphataemia Treatment of children and adult with X-linked hypophosphataemia Recommendations for management of burosumab in children Recommendations for management of oral phosphate and active vitamin D in children Recommendations for the prevention and managment of hyperparathyroidism Recommendations for recombinant human growth hormone Recommendations for musculoskeletal treatment Recommendations for managment of pregnant or lactating patients Recommendations for [...]
Endo-ERN is delighted to be a partner of The World Orphan Drug Congress Europe. This event is the largest and most established orphan drug & rare disease event worldwide. Meet over 2000 attendees, hear from 250 leading speakers, and connect with 130 exhibitors as we bring together experts from the start-to-finish of orphan drugs. From regulation and policy, to global pricing and gene therapy.
With this partnership we are delighted to inform you that Endo-ERN members are eligible for a free VIP pass. You can apply now.
The agenda is now available for download [...]
Another #raredisease webinar from Endo-ERN! Planned for Tuesday, 3, June 16:00-18:00. Register now and if you are unable to attend live you will receive a recording.
Congenital Adrenal Hyperplasia (CAH): Current guidelines and monitoring
Erica van den Akker and Sjoerd van den Berg (Rotterdam, The Netherlands)
Leukocyte Telomere Length in Children with Congenital Adrenal Hyperplasia
Ozair Abawi (Rotterdam, The Netherlands)
Predicting Treatment Outcome in CAH Using Urine Steroidomics and AI
Christa Flueck (Bern, Switzerland)
The effect of oral contraceptives in female adolescents with 21-hydroxylase deficiency: A prospective observational study
Claudia Boettcher (Bern, Switzerland)
This opportunity is open to a wide range of developers, including academics, individual researchers, startups, industry stakeholders, organisations from diverse settings, and patient-led initiatives. Support is available for both new device development and the adaptation of existing products to better serve paediatric and orphan populations.
Successful applicants will receive guidance from two dedicated innovation coaches to help advance their development goals. Assistance is offered across various stages of the product lifecycle, with tailored support in areas such as:
✔ Business and funding advice
✔ Network building
✔ Technical guidance
✔ Regulatory support
✔ Infrastructure for (pre-)clinical testing
✔ [...]
Save the date and register now!
After the success of the previous ISPAD/VAPES meetings local organizers (Varna Pediatric Endocrine Society, VAPES) aim to attract any interested pediatric endocrinologists, pediatricians, postgraduate trainees, medical students and nurses in 2025.
Topics covered at this event include:
Turner Syndrome McCune-Albright Syndrome Early-onset Autoimmune DiabetesYou can check out the complete programme including details of the international speakers and how to register.
An Endo-ERN endorsed event from member UZ Leuven – check out the full programme. REGISTER now or send an email for more information.
Organisers: Prof. Dr. Anne Rochtus, paediatric endocrinology & Marleen Jannis, administrative staff member
9:00: Welcome with coffee and tea Morning – Plenary sessions 09:20 Welcome (Prof. Anne Rochtus)
09:30 Prader-Willi syndrome from the past to the present (Prof. Annick Vogels)
10:00 Benefits of growth hormone for young and old (Prof. Anita Hokken-Koelega)
10:30 Overview of studies and new therapies (Prof. Anne Rochtus and Dr. Tessa Wassenberg)
11:00 Coffee [...]
Endo-ERN has endorsed an upcoming course by Endo-ERN HCP, Leiden University Medical Center (LUMC).
5th CAPITAL – June 5-6, 2025
You are invited to register for the 5th Care for Pituitary Adenomas Leiden (CAPITAL) multidisciplinary workshop in Leiden, The Netherlands, for two inspiring days of sharing best practices in pituitary care. We recommend attending CAPITAL together with all members of your multidisciplinary team, so you can discuss new insights and ideas during spare moments and reflect together on your own practice and future directions.
The previous editions of CAPITAL were highly interactive, with challenges and protocols shared, [...]
The European Commission is hosting workshops in several countries with a focus on patient rights in cross-border healthcare and European Reference Networks. Registration is now open for the Oradea covering Romania-Hungary workshop taking place 5 February 1000-1700 (EET).
Upcoming other events
28 February, 2025 Strasbourg
Contact DG Sante for more information.
