WELCOME: NEW ePAG Marta

Endo-ERN would like to welcome our newest ePAG Marta Baranska.

Marta is the founder and representative of the non-profit organization The Pituitary Gland Association Poland (Szczecin, Poland), which supports patients with pituitary gland disorders and their families.

 

Marta is living with Cushing’s Disease but balances taking care of her health with a busy and diverse career. Marta shares more, “I have built a strong career as a lecturer at the Maritime University, a sailing ship captain, and an expert at the International Maritime Organization in London. Additionally, I am a certified Cognitive Soul, Life & Business Coach.

Beyond [...]

Continue reading

This entry was posted in General.

CONTRIBUTE: Committee on Public Health (SANT) launches public consultation on rare diseases

A public consultation is now open seeking information from a range of #rare disease stakeholders. This information will be utilized by the Committee on Public Health (SANT) to bring a better understanding and detailed knowledge of the challenges of persons affected by rare disease and views of persons working with or involved in rare diseases.

Check out the full announcement here and complete the survey by the end of March.

Continue reading

This entry was posted in General.

CALL: Abstracts for ESPE-OSCAR Science Symposium, 18-19 September, Paris, France

Call for abstracts is now open for the upcoming European Society for Paediatric Endocrinology (ESPE) and French rare riseases healthcare network (OSCAR) being held 18-19 September in Paris France. Late-breaking abstract submission deadline 27 April, 2025, 23:59 CET. Abstract submitters will be notified regarding acceptance/rejection oral/poster by 26 may, 2025.

 

Check out the full programme with topics such as:

Short Stature and Bone Growth Lessons from the Growth Plate and Tools What’s New in Vitamin D? Teeth as a Model to Study Bone Pathology Fibrous Dysplasia of Bone Round Table on Recent Guidelines and Perspectives [...]

Continue reading

SUCCESS: TALENT meeting report

The 7th edition of the TALENT (Transition Adolescence and young aduLts – Endocrine diseases managemenT) conference took place on February 11-12, 2025, at the “Roma Eventi” congress center in Rome, Piazza della Pilotta 5. The event, officially endorsed by Endo-ERN, welcomed approximately 200 participants, including leading international experts in rare endocrine diseases.

 

A unique and longstanding feature of TALENT, present since its first edition, is its strong editorial activity, with dedicated working groups composed of both young and senior experts. These groups convene before the start of each conference to develop high-quality scientific papers focused on the key challenges [...]

Continue reading

This entry was posted in General.

WINNER: 2025 Patient Partnership Good Practice Challenge

Congratulations to Johan de Graaf and Petra Bruegmann for their recent win of the Eurordis 2025 Patient Partnership Good Practice Challenge! The aim of this initiative is to recogise outstanding examples of collaboration between patient representative and healthcare professionals within European Reference Networks (ERNs). This initiative has spotlighted innovative practices that enhance patient engagement and foster valuable partnerships between patients and healthcare professionals across the Networks, improving healthcare for people living with a rare or complex condition.

Johan and Petra are leading an initiative that aims to provide some light by mapping inequities in endocrine drug provision across Europe. The [...]

Continue reading

This entry was posted in General.

ACTIVE: ePAG Association GRANDIR

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

 

 

Continue reading

This entry was posted in General.

NEW: Materials from the European Commission for Rare Disease Day

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

 

Continue reading

This entry was posted in General.

NEW: Clinical Practice Recommendations for the Diagnosis and Treatment of X-linked hypophosphataemia

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 [...]

Continue reading

This entry was posted in General.

REGISTER NOW: World Orphan Drug Congress 2025

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.

 

27 October 2025 | Pre-Congress Workshops
28-29 October [...]

Continue reading