REGISTER: Orphan Medicines Development – ask the European regulator webinar

The EMA’s Orphan Medicines Office is hosting an interactive webinar on Rare Disease Day on 29 February 2024, to answer questions that developers of products for rare diseases or researchers in this field might have on orphan designations and rare disease development. The webinar will feature:

short presentations on the background of orphan designation and the benefits it has brought to patients; a live question-and-answer session enabling participants to ask their questions on orphan medicines development to a panel of regulatory experts.

More info: Orphan medicines development – ask the European regulator | European Medicines [...]

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REGISTER: EMA/ACT Training: Transitioning trials to the CTR (CTIS) for non-commercial sponsors

This training event aims to support non-commercial sponsors of clinical trials in transitioning their clinical trials that are expected to continue after 30 January 2025 from the Clinical Trials Directive to the EU Clinical Trials Regulation (Regulation (EU) No 536/2014). Moreover, the event will provide useful training in preparing new clinical trials following the Clinical Trial Regulation (CTR).

From 31 January 2025 onwards only the CTR and its Delegated Acts will apply. Sponsors, therefore, need to transition any trials that will continue after 30 January 2025 from the Clinical Trials Directive to the legal framework [...]

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REGISTER: ESE Talks…Congenital hypothyroidism and related problems

The next ESE Talks…webinar is Tuesday 6 February 2024, 17:00 – 18:15 CET

Chairs: Edward Visser (The Netherlands) and Athanasia Stoupa (France)

Fetal thyroid disorders

Dominique Luton (France)

Primary congenital hypothyroidism

Nadia Schoenmakers (UK)

Secondary congenital hypothyroidism

Paul van Trotsenburg (The Netherlands)

Panel/group discussion

Led by Edward Visser (The Netherlands) and Athanasia Stoupa (France)


Register now!

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OPPORTUNITY: PhD positions at Lubeck

Exciting opportunities are available at one of Endo-ERN’s reference centres!

Several PhD postions are available at the University of Lubeck’s Collaborative Research Center (CRC) 1665. Sexdiversity – Determinants, Meaning and Effects of Gender Diversity in Socialcultural, Medical and Biological Contexts is dedicated to researching the diversity of biological sex over the next four years.

Check out more information about CRC 1665 and the available positions.

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RDD EVENT: Ministry of Health Malta

On the occasion of the World Rare Disease Day, 29 February, 2024, the Ministry for Health of Malta, is hosting a one-day workshop on Cross-border Healthcare, Patients’ Rights and Rare Disease.

This workshop is being organised in collaboration with the European Commission and is being held under the auspices of the President of the Republic of Malta, His Excellency Dr. George Vella. Foreign and local speakers, including individuals with rare disease, will be coming together to share their personal experiences and provide updates on these important, under-recognized aspects of health care.


The agenda of the meeting and the connection [...]

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SAVE THE DATE: Joint Congress of ESPE and ESE 2025, Copenhagen Denmark

The European Society of Endocrinology (ESE) and the European Society for Paediatric Endocrinoogy (ESPE) are joining forces to create a unique joint Congress in 2025.

This very special joint Congress is themed Connecting Endocrinology Across the LIfe Course and will be held 10-13 May, 2025 in Copenhagen, Denmark.


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REGISTER: Training on strategies to foster solutions of undiagnosed rare disease cases

As part of the training activities proposed by EJP RD, an 3-day training course on Training on strategies to foster solutions of undiagnosed rare disease cases is being organised by Istituto Superiore di Sanità (ISS) in close collaboration with EJP RD partners. The training will be held in Rome, Italy on 13-15 March 2024.

Through the presentation of sample use cases that have long eluded diagnosis, the course will provide participants with with useful tools, instruments and knowledge on novel strategies to foster solutions of undiagnosed rare diseases cases. Moreover, the course will facilitate networking among professionals [...]

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REGISTER: The differential diagnosis of discrepant thyroid function test webinar

Endo-ERN experts will present a case-based discussion on Tuesday, 23 January at 1500-1600 CET.

Chairman: Prof Luca Persani, Istituto Auxologico Italiano – Istituto di Ricovero e Cura a Carattere Scientifico, Italy

Speakers Dr Irene Campi, Istituto Auxologico Italiano – Istituto di Ricovero e Cura a Carattere Scientifico, Italy and Dr Edward Visser, Erasmus MC, University Medical Center Rotterdam, the Netherlands

1500-1515 Brief introduction Prof Luca Persani

1515-1545 Case-based discussion with Dr Campi and Dr Visser

1545-1600 Q&A


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UPCOMING: 12th European Conference on Rare Diseases & Orphan Products (ECRD)

The ECRD is the largest, patient-led rare disease policy-shaping event held in Europe. This event will take place 15-16 May, 2024 at The Square in Brussels.

By bringing together people with rare diseases and patient advocates, policy makers, healthcare industry representatives, clinicicans, regulators and Member State representatives, EURORDIS harnesses the power of this extensive network to shape goal-driven rare disease policies of the future.


Check out the programme for more information.


Interested in submitting an abstract? The deadline is 23 February and more details can be found here.

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