ERDERA Clinical Trial Call is now open

ERDERA has opened its Clinical Trial Call (ECTC) to support multinational, GCP‑compliant Phase I, Phase I/II and Phase II interventional clinical trials in rare diseases. This call aims to generate robust clinical evidence and, where appropriate, data of regulatory relevance to support future regulatory interactions and subsequent clinical development. It is designed for rare disease trials where patient populations are […]

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SSIEM 2026 Annual Symposium in Helsinki

News, Events, Networks

The Annual Symposium of the Society for the Study of Inborn Errors of Metabolism will be held in Helsinki, Finland, from 25 to 28 August 2026. This year’s theme of the symposium will be the “Next Generation Metabolic Medicine”, inviting participants to reflect on where research in the IMD field is heading and how today’s science shapes

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International Glycogen Storage Disease Conference 2027

Events, News

Save the date for the International Glycogen Storage Disease Conference (IGSD 2027)! The conference will be held from 17 to 19 June 2027 at the University Medical Center Groningen (UMCG) in the Netherlands, situated in the heart of the beautiful and vibrant city of Groningen. During the conference there will be opportunities for social and networking

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2nd Patient Meeting & Scientific Conference – CDG at the Children’s Memorial Health Institute (CMHI)

Events, News, News for Patients

“CDG at the CMHI” is a two-day event bringing together the scientific community, clinicians, patients, and their families, which will take place on May 29–30, 2026, at the Children’s Memorial Health Institute (CMHI) in Warsaw, Poland. The first day of the event is dedicated to lectures and expert discussions on the diagnosis, symptoms, and modern treatment

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18th International MPS 2026 Symposium & MPS MasterClass

Uncategorized, Education, Education for Patients, Education for Professionals, Events, News

The 18th International Symposium on Mucopolysaccharidoses and Related Lysosomal Diseases will take place on June 4-7, 2026, in Florence, Italy at Palazzo Affari and Palazzo dei Congressi. This global event will reflect on the last 20 years of progress made in Mucopolysaccharidoses (MPSs) and set sights on the next 20 years of innovation, advocacy, and collaboration.

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call for applications for Affiliated Partner within the European Reference Networks

News, Networks, Other

The European Commission has launched the 2026 Call for Applications for Affiliated Partners within the European Reference Networks (ERNs). The call is designed to strengthen cross-border collaboration and improve access to highly specialised care for patients with rare, low-prevalence and complex diseases. Applications will be accepted from 13 April 2026 until 1 September 2026, 23:59 CET. Why this call matters ERNs bring together European hospital

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high-level conference: Patients’ rights on cross-border healthcare and the European Reference Networks

Events, Networks, News, News for Patients

The hybrid high-level conference on “Patients’ rights on cross-border healthcare and the European Reference Networks” will take place on 26 March 2026. This high-level conference will focus on the EU’s achievements in making cross-border healthcare more accessible, as well as at the barriers to making it more widely known and used. The European Commissioner for Health

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SIMMESN & MetabERN Collaborative Success: Survey Results on Diagnostic and Monitoring Activities for Patients with Metabolic Disorders are Available

News, Events, Networks, News for Patients

We are proud to share the results from a crucial survey on diagnostic and monitoring activities for patients with metabolic disorders, a collaborative effort between Società Italiana per lo Studio delle Malattie Metaboliche Ereditarie e lo Screening Neonatale (SIMMESN) and MetabERN Italy, led by Prof Carlo Dionisi Vici (Bambino Gesù Children Hospital, Rome, Italy). This initiative

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Essential biomarkers in IEM: basic course

News, Education, Education for Professionals

Enrol in the online course “Essential biomarkers in IEM: basic course” provided by the SJD Education of the Sant Joan de Déu University Hospital in Barcelona, starting on 11 March 2026. The course is delivered as a series of six focused webinars, each centred on a key biochemical presentation. These sessions bring together leading international experts in

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“The Classical Galactosemia European Care Pathway” is now available

Resources, Networks, News

MetabERN is pleased to inform you that “The Classical Galactosemia European Care Pathway” is now available. This initiative was spearheaded by Mrs Maaike van Kempen, Mrs Clementine Bry, Prof. Estela Rubio-Gozalbo, Prof. Eileen Treacy, and Prof. Annet Bosch in collaboration with JARDIN Joint Action. The pathway is built upon the International Clinical Guideline for the Management of

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Rare Disease Day 2026 – More than you can imagine!

News, Events, News for Patients, Press Releases

The last day of February is a special occasion dedicated to raising awareness about rare diseases. Rare Disease Day aims to shine a light on these conditions and to advocate for improved access to treatment and care for the millions of individuals and families affected by these diseases. In the European Union, a rare disease is

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