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we hope you’ve had a pleasant start to 2026 and are ready to make this year even better than the last!
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We begin the year with exciting news: the High-Level Meeting on a European Innovation and Care Ecosystem for Rare and Complex Diseases has culminated in a Declaration to build a sustainable research and innovation ecosystem, supported by dedicated funding. We, together with many other European Reference Networks, have signed this Declaration and will actively work to advance its goals.
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What else is new in January? Discover our new patient journey on Rare Neurological Diseases, providing an overview of common experiences shared across these conditions. Watch the video interview with our member Belen Dueñas Perez, who shares insights into her project on myoclonus dystonia and her work in developing clinical guidelines. Also, a training on Data, Ethics and AI by EURORDIS as well as outreach materials for Rare Disease Day, designed to help raise awareness and engage your community.
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And as always you will find information on our upcoming webinars and events. Enjoy reading!
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Best wishes,
The ERN-RND Coordination team
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CONTENT
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About us
Disease Knowledge
ERN-RND Webinars
Cross-border Healthcare
Data Ethics and AI Training
Rare Disease Day 2026
Upcoming Events
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ABOUT US
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HIGHLIGHT Declaration on the European Innovation and Care Ecosystem for Rare and Complex Diseases
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Last December's High Level Meeting on a European Innovation and Care Ecosystem for Rare and Complex Diseases brought together policymakers, researchers, healthcare professionals, industry and the patient advocacy to help shape the future of rare disease policy in Europe.
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This collective effort culminated in the Declaration on the EU Innovation and Care Ecosystem for Rare and Complex Diseases - a bold commitment to building a sustainable research and innovation ecosystem, supported by dedicated funding. We, alongside many other European Reference Networks, have signed this Declaration and will actively work towards advancing its goals within our remit.
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EURORDIS Black Pearl Holistic Care Award for Bulgarian Huntington Association (led by our ePAG Natalia Grigorova)
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This center was founded in 2015 by our ePAG Natalia Grigorova - congratulations on this huge achievement! Learn more about the work of the organization here.
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Myoclonus Dystonia - Interview Belén Dueñas Pérez
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Myoclonus Dystonia often begins in childhood, yet it is frequently mistaken for more common conditions such as cerebral palsy—delaying accurate diagnosis and effective care. To change this, a study among ERN-RND centers across Europe was conducted in order to raise awareness, strengthen genetic diagnosis, and better understand how the condition is managed.
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The results of this study now lay the groundwork for clinical treatment guidelines for the disease. Learn more about this condition and treatment options in our video interview with ERN-RND member Belén Dueñas Pérez.
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DISEASE KNOWLEDGE
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NEW Introducing the Patient Journey for Rare Neurological Diseases
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This new Patient Journey provides an overview of the common experiences shared across the rare neurological diseases covered by ERN-RND. After developing disease-specific journeys for various conditions, it became clear that many challenges and experiences are shared across these diseases. To reflect this, our ePAGs and clinicians consolidated these insights into a Patient Journey for Rare Neurological Diseases.
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This shared journey is designed to support both clinicians, by guiding their interactions with patients, and patients themselves, helping them better understand and navigate their own path.
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You can find the English version of the Patient Journey here, free for download. Translations into other European languages are currently in progress, with Portuguese and Polish already available.
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ERN-RND WEBINARS
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Upcoming Joint Educational Webinars
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ERN-RND provides free educational webinars on rare neurological and neuromuscular diseases - in collaboration with the European Reference Network for Rare Neuromuscular Diseases (EURO-NMD) and the European Academy of Neurology (EAN). The goal is to share knowledge on rare neurological, movement and neuromuscular disorders via a series of webinars presented by expert members of both networks.
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TODAY
15.01.2026, 4-5 pm CET | ‘Progress in Genetic Diagnosis for Neuropathies’, by Andrea Cortese, University College London, Department of Neuromuscular Diseases, UK. SIGN UP here. (EURO-NMD)
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22.01.2026, 5-6 pm CET | ‘Biomarkers and Outcome Measures for Clinical Trial Readiness in CMT’, by Mary Reilly (University College London, Department of Neuromuscular Diseases, UK). SIGN UP here. (EURO-NMD)
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29.01.2026, 4-5 pm CET | ‘Current Progress in Charcot-Marie-Tooth disease (CMT) Treatments’, by Kleopas Kleopa, Cyprus Institute of Neurology and Genetics (CING). SIGN UP here. (EURO-NMD)
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03.03.2026, 3-4 pm CET | ‘Getting the most out of Physiotherapy in Dystonia’ by Maarten Nijkrake, Radboud University Meidcal Center, Nijmegen, the Netherlands. SIGN UP here. (ERN-RND)
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eanCampus offers monthly webinars, 2 master classes per year and weekly podcasts on 3 expert levels (basic, advanced, expert), in total more than 1000 pieces.
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Free Courses for Healthcare Professionals, Caregivers & Family Members
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The Huntington Academy is a multinational, multilingual initiative designed to address the significant gaps in care provision for families impacted by Huntington’s disease (HD). Its mission is to empower the HD community through knowledge, skills, and collaborative learning, fostering improved care, advocacy, and quality of life.
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It is an innovative and comprehensive e-learning platform designed to provide accessible and high-quality educational resources for both formal (healthcare professionals) and informal (family members and friends) caregivers of individuals affected by HD. The Huntington Academy contents are available in four languages – Bulgarian, English, French and Spanish.
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The platform includes two transversal core courses — “What is HD? (HD basics)” and “Communication Skills in HD Care” — and seven domain-specific courses covering key disciplines in HD care (Neurology, Psychology, Nutrition, Physiotherapy, Speech Therapy, Occupational Therapy and Oral Care).
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All the Huntington Academy contents were jointly created by patient organizations, family members and healthcare professionals from the countries involved in the consortium.
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AGI Webinar Series: Preventive Trials for SCAs
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Ataxia Global Initiative launched a new webinar series focusing on preventive trials in the spinocerebellar ataxias (SCAs) — a developing area in ataxia research.
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Preventive approaches build on recent progress in understanding pre-ataxic disease stages and early biomarkers, representing the next step toward earlier intervention. This concept has already been explored in other neurodegenerative diseases, and the series aims to translate these insights to SCAs. See the full program here.
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The webinars will address:
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• The rationale for preventive trials
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• Characterization of the pre-ataxic stage in SCAs
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• Lessons learned from related conditions
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• The potential of emerging biomarkers
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CROSS-BORDER HEALTH CARE
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MLD Treatment Eligibility Panel
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In cooperation with the MLD initiative, ERN-RND established a standard pathway for consulting an international MLD expert panel whenever possible benefits of treatment with hematopoietic stem cell transplantation or gene therapy are not straightforward for a patient with confirmed MLD diagnosis. Upon submission of an eligible case, the treatment eligibility panel is convened on an ad hoc basis, organized and supported by the ERN-RND CPMS helpdesk.
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We encourage all physicians in Europe to discuss the possible benefits of treatment options for difficult cases with this MLD expert panel.
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You would like to discuss one of your patients with the panel?
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Online Multidisciplinary Board for Deep Brain Stimulation in Dystonia
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ERN-RND has established a standard pathway for providing multidisciplinary expert recommendations concerning Deep Brain Stimulation (DBS) in Dystonia.
Potential questions which the multidisciplinary board may address include (but are not limited to):
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- INDICATION DBS candidate yes/no?
- TARGET Which Target; GPi, STN, VoA?
- PROGRAMMING Treatment advices of implanted patients
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To make use of this opportunity, relevant medical data must be provided via the telemedicine platform CPMS beforehand.
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Advice will be provided by at minimum one dystonia expert specialized in DBS and a stereotactic surgeon specialized in DBS, plus further specialists invited case-by-case in a virtual meeting with the referring clinician.
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Neuroradiology Expert Advice for Diagnostic and Management Decisions in RND
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ERN-RND has established a standard pathway for providing expert neuroradiological advice across sites. Our ERN-RND member hospitals (and affiliated partners) are encouraged to contact us for any RND case for which they would like a second opinion as a basis for individual recommendations for diagnostic and management strategies to optimize patient care.
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Possible scenarios for which clinicians might want to use this offer include (but are not limited to):
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- Child with mild developmental delay: benign / onset of disease? -> cMRI: delayed brain maturation vs. (subtle) structural abnormalities
- Child with delay + subtle signs: acquired / genetic? -> cMRI: acquired disease, e.g. CMV infection / TORCH vs. likely genetic
- Acutely ill patients with suspected encephalopathy -> cMRI: secondary, e.g. infectious / para-infectious vs. metabolic / genetic
- Cerebellar syndrome with fast progression -> cMRI: secondary, e.g., cerebellitis vs. genetic
- Patient after pharmacological treatment showing brain volume reduction -> cMRI: side effects vs. neurodegenerative / neurogenetic disease
To make use of this opportunity, imaging data must be provided via CPMS. Advice will be provided in a virtual meeting between the referring physician plus the initial neuroradiologist and a dedicated ERN-RND neuroradiological expert clinician (and further experts if necessary. Meetings are facilitated by the ERN-RND CPMS helpdesk.
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You are affiliated to an ERN-RND expert center and would like to receive neuroradiology advice for one of your patients?
Please contact us.
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Learn more about the Neuroradiology Expert Advice Panel in this video interview with Eva Bültmann, who is an expert in neuroradiology and coordinates this panel.
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RARE DISEASE DAY 2026
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Rare Disease Day 2026 - all about Equity
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Less than 6 weeks till Rare Disease Day (RDD) - help us raise awareness for the 300 million people worldwide living with a rare disease, their families and carers. And we are looking forward to the RDD campaign from JARDIN, where you will find us and other ERNs!
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Also: go get the RDD outreach material (email signature, virtual background, social media banner...) to support the rare disease community - also in many different languages. Find it here.
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And if you have an event going on post it on the RDD website!
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Watch the official RDD 2026 video here.
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EURORDIS: Call for Applications on Data, Ethics and AI training
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Applications are now open for the 2026 Data, Ethics and AI training developed by EURORDIS and ERDERA, offering high-quality training for rare disease patient advocates and early-career researchers (PhD candidates and up to two years post-PhD).
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This training has been developed in response to the growing need for advocates and researchers to understand and navigate the complexities of data management, ethical considerations, and the role of AI in rare disease research across Europe.
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The online part of the training will run from March to July 2026, followed by the in-person sessions on 14–15 September 2026 in Barcelona.
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UPCOMING EVENTS
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3rd Expert Summit on the Future of Deep Brain Stimulation
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February 22-24, 2026, Würzburg (Germany)
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- Network-wide modulation and therapeutic effects of DBS
- Translational approaches in DBS
- Computational modelling and imaging in DBS
- Hardware innovations and technological needs
- Digital Innovations in DBS
- Closed-loop-neurostimulation
- Alternative technologies for targeted neuromodulation
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Find the programme here and more information here.
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13th European Conference on Rare Diseases and Orphan Products
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June 3-4, 2026, Prague and online
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The next ECRD will be held on 3-4 June 2026 in Prague and will be titled “Rare Diseases in a Changing & Competitive Europe: Shaping policies to address the unmet needs of people living with rare diseases”.
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38th Annual Meeting of EACD
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June 3-6, 2026, Galway, Ireland
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The old Irish wisdom ‘Mol an Óige agus Tiocfaidh Siad’ – Encourage the young and they will flourish – is the theme of EACD 2026. The conference will focus on disability and health through a holistic lens. Taking a lifespan approach and positioning the voice of people with childhood-onset disabilities and their families at the centre of all that we do, we aspire to support people with disabilities to flourish not just now but into their futures.
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Our ePAG Mary Kearney will also give a talk on "From Survey to Practice: Strengthening Transition Pathways in Rare Disorders to Foster Lifelong Participation, Community Integration, and Person-Centred Continuity of Care"
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12th Congress of the European Academy of Neurology
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June 27-30, 2026, Geneva, Switzerland
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EAN's overarching theme for 2026 is Brains, Bytes & Beyond: Tech in Neurology. The outsourcing of cognitive capacities from humans to artificial intelligence offers huge opportunities but also significant challenges and risks. In clinical neurology, the impact of computing will range from applications like restorative therapies to the very fundamentals of our profession such as clinical reasoning and decision making. Through this year’s theme, EAN will explore the extent to which computing is finding its way into neurological practice.
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Early bird registration is open, click here. Find the programme here.
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ICAR 2026
November 10-13, 2026, Atlanta, Georgia (USA)
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Ataxia UK, National Ataxia Foundation (NAF), Friedreich’s Ataxia Research Alliance (FARA), and Ataxia Global Initiative (AGI) are pleased to announce the date for the next International Congress for Ataxia Research (ICAR).
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ICAR 2026 will take place at the Renaissance Atlanta Waverly Hotel & Convention Center in Atlanta, Georgia, U.S. Please save the date for November 10-13, 2026.
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ICAR 2026 will be the place to share the latest ataxia research, including updates on Friedreich's ataxia and SCAs 1, 2, 3, 6, 7, and more. Attendees will hear developments in novel treatment approaches, clinical trial results, and scientific debates from leading ataxia researchers. There will also be the opportunity to network with academic and industry leaders. Special sessions and events are planned for junior researchers.
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Registration and abstract submission will open in March.
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NEWSLETTER
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The ERN-RND Newsletter is published monthly. Please forward this newsletter to your colleagues who can also sign up to receive it directly here or by emailing communication@ern-rnd.eu
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Thank you for your support and take care.
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ERN-RND Coordination team
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