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 [...]

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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 [...]

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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

 

 

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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

 

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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 [...]

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CALL OPEN: DeCODe project call for support to assist devleopers of paediatric and orphan medical devices

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

✔ [...]

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REGISTER: Upcoming Workshops Patient Rights and Rare Diseases in the European Union

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.

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COMPLETE: Endo-ERN Transition of Care Working Group Survey

The Endo-ERN Transition of Care Working Group, invites you to complete this survey.

The survey aims to gather valuable insights into the current practices, challenges, and opportunities in managing the transition of care for patients with rare endocrine diseases, focusing on both pediatric and adult care perspectives.

Who should complete the survey?

Healthcare professionals directly involved in transition care for patients with rare endocrine conditions, from both pediatric and adult sides.

Time required: Approximately 15–20 minutes.

Dissemination Request

We kindly ask that the survey link be shared with all Endo-ERN members, including Reference Centres and associated [...]

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REGISTRATION: ERDERA 2025 Joint Transnational Call for Proposals Info Webinar and Resources

The European Rare Diseases Research Alliance (ERDERA) Joint Transnational Call (JTC) for Proposals 2025 is now open! Submissions are invited from research teams from across Europe and beyond to submit collaborative projects focused on “Pre-clinical therapy studies for rare diseases using small molecules and biologicals – development and validation”.

This JTC will build upon the significant progress made by previous European Joint Programme on Rare Diseases (EJP RD) calls, with the aim of fostering international partnerships to develop innovative therapies for rare diseases. National and regional funding bodies from over 30 countries, including France, Belgium, Germany, Italy, and all Baltic [...]

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POSTER WINNER: Endocrine Medicine Shortages

Endo-ERN ePAGs Johan de Graaf, Dutch Pituitary Foundation and Petra Brügmann (EMENA) with Endo-ERN colleague Emily White (Endo-ERN) and Dirk de Rijdt of the European Society Endocrinology prepared a poster Multistakeholder Mapping of Endocrine Medicine availability and shortages in Europe: Endo-ERN expert centres.

This poster was Best Poster at the European Rare Disease Research Coordination and Support Action consortium (#ERICA) at the first ERICA ERN Research Conference.

 

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