19th ERN GUARD-Heart Board Meeting 19 March 2026 in Bucharest, Romania

All Board members have received the invitation to register in the upcoming ERN GUARD‑Heart Board Meeting. Participation is foreseen  one representative per HCP, as well as two ePAG delegates. For those unable to join on site, an online option will be available. The meeting will take place at the: Bucharest Courtyard by Marriott Floreasca Hotel Bucharest, Romania 
Any questions contact us ♥ 

Hereditary Cardiovascular Diseases Course 20-21 March 2026, Bucharest, Romania

Register and join this course, where national and international experts will present highly relevant and up‑to‑date topics in the field.
Held under the auspices of the Romanian Society of Cardiology, UMF “Carol Davila”, the Expert Center for Rare Genetic Cardiovascular Diseases, and ERN GUARD‑Heart♥.

Please: sign the Declaration on a European Innovation and Care Ecosystem for Rare and Complex Diseases

Dear ERN- representatives,
We are excited to invite you to officially endorse and sign the Declaration on a European Innovation and Care Ecosystem for Rare and Complex Diseases (sign p.17) Launched at the first-ever High Level Meeting for Rare and Complex diseases (HLM Rare) in December 2025, this Declaration marks a pivotal moment for rare disease care and innovation across Europe, building on a vision to strengthen the collective impact of the European Reference Networks♥.

Who are our ePAGs?
Céline’s Journey: Perseverance, Resilience, and Hope 
Family’s Story and Commitment to AMRYC (Association for Hereditary Cardiac Rhythm Disorders)

This deeply personal story is shared by Sophie Pierre, ePAG representative and vice‑president of AMRYC (France), an association supporting patients with inherited cardiac rhythm disorders such as long and short QT syndromes, Brugada syndrome, Catecholaminergic Polymorphic Ventricular Tachycardia, and early repolarization syndrome. Sophie recounts the journey of her daughter Céline, diagnosed in early infancy with the rare cardiac rhythm disorder short QT syndrome type 2, identified by a dedicated inherited arrhythmia specialist at Necker Hospital in Paris from the French Consortium.
 Through medical challenges, additional health complexities, and family hardships, their story shines with hope, determination, and unwavering commitment. Their experience has not only shaped their lives but also inspired deep involvement in AMRYC, supporting other families living with rare inherited cardiac rhythm disorders and advocating for awareness, preparedness, and better care♥.

Timothy Syndrome Alliance: Upcoming CACNA1C Conference 2026 

We are pleased to share an announcement from ePAG‑linked organisation Timothy Syndrome Alliance (TSA), a charity supporting individuals with CACNA1C‑related disorders. These rare genetic conditions are associated with multisystem clinical features, including neurological symptoms (hypotonia, epilepsy, seizures), neurodevelopmental conditions (intellectual disability, developmental delay, autism, ADHD), cardiac abnormalities, and syndactyly.

TSA will host a two‑day in‑person conference in Cardiff, UK, on 22–23 July 2026, bringing together patients, families, clinicians, and researchers to co‑develop a patient‑prioritised research agenda.

Registration is open for patients, families, researchers, and clinicians, and abstract submissions are welcomed until 16 February 2026♥.

Generated Documents for ERN GUARD‑Heart Registries

The ERN GUARD‑Heart coordination team, in collaboration with the Legal Office of Amsterdam UMC, has developed a template for a Joint Data Registry Agreement (JDRA). This template is an adapted version of the agreement developed by the Dutch Federation of Universities and is intended to facilitate arrangements regarding governance, data sharing, and data access for multicentre patient registries.

The template has been specifically designed for use within ERN GUARD‑Heart and can be used by centres that wish to establish a registry within the ERN and make use of the coordination and data‑hosting services provided by Amsterdam UMC.
The JDRA consists of several appendices that need to be completed and attached♥.

Sudden Cardiac Death Letter for Families
– Call for Translations

The letter for families following the (young) sudden cardiac death of a loved one, which was created by the E-PAGs members and approved by ERN experts, is translated in French. It would be great to have a translation in other European languages as well. The letter can be shared with hospitals, emergency physicians, paramedics, coroners, pathologists, family doctors, paediatricians, and others involved in sudden cardiac death in the young. Please contact the ERN-coordination office if you can help with the translations. The letter is available on the website♥.

Medical Device Shortages: Watch the Cross-ERN Webinar on Regulatory Challenges

On 21 January 2026, ERN eUROGEN hosted a joint webinar with ERNICA, ERKNet, and ERN GUARD-Heart to address a growing concern across rare and complex disease care: medical device shortages and regulatory constraints.

The webinar explored the real-world impact of reduced device availability, evolving EU regulatory requirements, and challenges around off-label and compassionate use of medical devices.
Expert speakers (Elena Arbelo, Thomas Krasemann, Marc Gewillig from ERN GUARD-Heart)shared clinical, regulatory, and industry perspectives, highlighting how these issues affect patient access to life-saving technologies and the delivery of specialised care across Europe. The discussion also examined the current status of Medical Device Regulation (MDR) registrations and ongoing advocacy efforts within and beyond the ERN framework.
This cross-ERN collaboration provides valuable insights for clinicians, researchers, patient representatives, and policymakers working in rare disease care, and remains highly relevant as regulatory and procurement challenges continue to evolve ♥. 

REMEDi4ALL Drug Repurposing Bootcamp for Academics – Applications Open

We are pleased to share a learning opportunity that may be of interest within the Network. There are still places available for the REMEDi4ALL Drug Repurposing Bootcamp for Academics, taking place 11–12 March 2026 at Hinxton Hall, Wellcome Genome Campus (Cambridge, UK).

This in‑person training is designed for early‑career researchers and principal investigators who are leading, or planning to lead, a drug repurposing project. The programme supports effective translation of repurposing research through expert lectures, interactive discussions, and small‑group working sessions, and offers the chance to engage with key stakeholders in the field.

Participation is fully funded: course fees, travel, and accommodation are covered for selected applicants.
Places are limited to 20, and applications close on 25 February 2026♥.

Registration Now Open for ECRD 2026 – European Conference on Rare Diseases & Orphan Products

Registration is now open for ECRD 2026, taking place on 3–4 June 2026 in Prague, Czech Republic.
ECRD is Europe’s largest patient‑led conference dedicated to shaping the future of rare disease care, research, and policy. It brings together clinicians, researchers, patient organisations, and stakeholders across sectors to advance solutions that improve the lives of people living with a rare disease.
The overarching theme of ECRD 2026 is:
“Rare Diseases in a Changing & Competitive Europe: Shaping policies to address the unmet needs of people living with rare diseases.”
ECRD 2026 will be a key milestone—five years after Rare2030 and midway to the WHO Global Plan 2028 target. The event will gather the rare disease community to assess progress and work collectively toward an EU Action Plan (or Strategic Framework) for Rare Diseases, often described as “planning for a Plan.”
Participants who register before 26 February 2026 will benefit from exclusive Early-Bird rates♥. 

RealiseD Webinars: Innovation in RD Clinical Trials

The RealiseD project is hosting a four-part webinar series exploring how to improve clinical trial design and evidence generation in rare and ultra-rare diseases. The series brings together experts from research, regulation, industry and patient organisations to address key challenges, including complex trial methodologies and the need for more patient-centred approaches.
 Funded by the Innovative Health Initiative, RealiseD aims to establish new standards that support innovation, reduce inequalities, and improve patient access to effective therapies♥.

Latest ERN GUARD-Heart Publications

Here is an overview given of scientificLatest ERN GUARD-Heart Publications publications related to ERN GUARD-HEART. Publications were obtained from the PubMed database. All publications where one of the authors has an affiliation link where “ERN GUARD-HEART” is mentioned, are screened and added to the publication overview♥.