Welcome
Welcome to the Neurogenetic Winter School – Budapest 2026
We are delighted to welcome you to the Neurogenetic Winter School to be held in Budapest in December 2026. This meeting is organized with the active participation of members of the EAN Neurogenetics Scientific Panel, the EAN Muscle & Neuromuscular Junction Scientific Panel, and the Rare Diseas Scientific Coordinating Panel. Our goal is to create an inspiring educational environment for clinicians, researchers, and trainees interested in the rapidly evolving field of neurogenetics.
In recent years, advances in genetics and molecular medicine have fundamentally transformed our understanding of neurological disorders. At the same time, the emergence of innovative therapeutic strategies—including gene-based and molecularly targeted treatments—has opened entirely new possibilities for patients with previously untreatable conditions. The Neurogenetic Winter School aims to provide participants with a comprehensive overview of these developments while fostering discussion about the opportunities and challenges that accompany them.
The program will focus on therapeutic perspectives in neurogenetic diseases. Beyond discussing cutting-edge and emerging therapies, we will address the essential role of real-world data in understanding treatment effectiveness and long-term outcomes. Monitoring disease progression and treatment response has become increasingly important in the era of disease-modifying therapies, and therefore special attention will be given to patient follow-up strategies, clinical outcome measures, and the development and validation of biomarkers.
In addition, the Winter School will explore the regulatory and translational challenges that accompany the introduction of innovative treatments for rare neurological diseases. Bringing therapies from laboratory discovery to clinical implementation requires close collaboration among clinicians, scientists, regulatory authorities, industry partners, and—most importantly—patients and patient organizations.
Through lectures, case discussions, and round-table discussions this course aims to create a dynamic platform for learning, collaboration, and exchange of ideas among participants and faculty. We hope the meeting will not only deepen knowledge in neurogenetics but also stimulate new collaborations and inspire the next generation of clinicians and researchers working in the field.
We look forward to welcoming you to Budapest for an engaging and stimulating Winter School dedicated to advancing the future of neurogenetic care and research.
On behalf of the organizing committees
Mária Judit Molnár
Location
Venue and gallery
Danubius Hotel Hungária
Danubius Hotel Hungária
Address: H-1074 Budapest,
Rákóczi street 90.
https://danubiushotels.com/hungaria
Committees
Mária Judit Molnár
MD, PhD, Professor of Neurology, Semmelweis University
Program
Scientific program
Friday, 4 December, 2026
Registration opens
Welcome address
Neurogenetic Diseases: Bridging Genetic Insights with Clinical Advancements
F. Magrinelli
Session 1: Muscle, Nerve, Beyond: Innovative Therapies in Neurogenetic Disorders
Genetic Therapies in Duchenne Muscular Dystrophy: Progress and ChallengesM.J. Molnár
Beyond Survival: How SMA Treatment and Monitoring Are Being TransformedJ. Haberlova
Emerging Therapies in Pompe Disease: Gene, Enzyme, and Molecular StrategiesA. Toscano
From Gene to Treatment: Therapeutic Advances in TTR Amyloidosis and Fabry DiseaseZ. Grosz
Neurofibromatosis Type 1: From Genotype to Multisystem ManagementM. Szegedi
Lunch break
Session 2: Neurodegenerative Diseases and Emerging Therapies
Novel disease modifying therapies for Huntington’s DiseaseJ.M. Burgunder
Treatment approaches of hereditary mitochondrial optic neuropathiesC. La Morgia
Diagnostic and management of MELAS M. Mancuso
Innovative Strategies for Managing Leukoencephalopathy: Gene Therapy and BeyondA. Federico
Neurodegenerative Seizure Disorders and Genetic Epilepsies: The paradigmatic case of Neuronal Ceroid LipofuscinosisS. Della Vecchia
Current Therapies and Future Perspectives in Amyotrophic Lateral SclerosisM. Filosto
Coffee break
Session 3: Bring Your Own Cases: Interactive Session
Short presentations (6) from participants (10 minutes + 2 minutes for questions) on their own experience in managing neurogenetic disorders
Saturday, 5 December, 2026
Registration opens
Session 4: Inherited Ataxias and Movement Disorders
Friedreich’s Ataxia: Current Treatments and the Promise of Gene TherapyE. Indelicato
Emerging Treatment Strategies for Spinocerebellar Ataxia and Other Genetic Ataxias C. Mariotti
Neurostimulation therapies in neurogenetic disordersE. Moro
Invasive therapies in neurogenetic disordersD. Zadori
Session 5: Monitoring Disease Progression and Treatment Efficacy
Data-Driven Monitoring of Progression and Treatment Efficacy in Inherited Neurological DisordersP. Balicza
Biomarkers for Hereditary Movement DisordersJ. Faber
Biomarkers for FSHDS. Sacconi
Advanced digital biomarkers in neuromuscular disordersG. Siciliano
Lunch break
Session 6: Clinical Trials and Drug Development in Neurogenetic
Navigating Regulatory Pathways for Genetic TherapiesG. Palomo Carrasco
Challenges in Clinical Trials for Neuromuscular DiseasesM. Mroczek
Federated Data Sharing: Accelerating Drug Development and Regulatory ApprovalV. Molnár
Panel discussion: The Path Forward: Integrating Genetic Therapies into Clinical Practice
Overview: Interactive panel discussing the future of genetic therapies, challenges in clinical integration, regulatory hurdles, and the role of patient advocacy.
Closing remarks of the Winter School
Lunch break
Session of the Hungarian Society of Clinical Neurogenetics (Hungarian session)
J. Boczán
Call for abstract to Interactive Case Discussion
The Interactive Case Discussion session can accommodate up to eight short presentations. The Scientific Committee will select cases for oral presentation based on submitted proposals, while additional cases can be presented as posters.
Abstracts should be prepared and submitted according to the instructions below:
- Language of the submitted abstract: English
- Title of Presentation
- Full name of author(s), (Please write the full name for all co-authors, do not use initials)
- Affiliation of author(s)
- In case of multiple authors / affiliations, please use index numbers in superscript
- The maximum length of the summary is one A/4 page
- MS Word file, Times New Roman font type, 12 pts font size, single line spacing required
- Text files should not contain headers or footers
- Please avoid abbreviations, if used, please elaborate it at the first time
- Abstract template
Registration
Pre-registration is available in the conference’s official online database. Please register as soon as possible so that we can keep you informed about our latest news. We kindly ask you to use the online registration system below.
2 November, 2026. | Deadline for payment of the discounted registration fee.
Delegate registration fee
190 EUR
215 EUR - late registration
- Access to the scientific sessions
- Admission to the exhibition
- Coffee breaks
- Lunches
- Welcome dinner on 4 December, 2026
- Winter School material
- Admission to the MKNT* Conference on 5 December, 2026
- Electronic certificate of attendance
- VAT
* Magyar Klinikai Neurogenetikai Társáság 2026. évi konferenciája (magyar nyelvű) | Annual Conference of the Hungarian Society of Clinical Neurogenetics (Hungarian session)
Terms and conditions of participation
Advanced online registration is a condition of participation in the conference. During the online registration you should accept the terms and conditions of participation as well as the terms of cancellation written on this website. The registration fee includes entertainment allowances, such as catering services, reception, dinner etc. In the final invoice the amount of these services will be indicated as intermediary service. See also the general terms and conditions.
Cancellation policy of registration
All the cancellations and changes must be sent to Diamond Congress Ltd. in written form.
Refund of the registration fee will be made as follows:
- 100% refund – in case of cancellation received before 10 May, 2026
- No refund – in case of cancellation, received after 10 May, 2026
Payment
All payments should be made in HUF. All charges due to bank transfers have to be paid by the sender. We advise you to bring the proof of payment with you when registering for the event in Budapest.
Bank transfer has to be made to
Bankszámlaszám:
Diamond Congress Kft. – MET2026
Bank: Raiffeisen Bank Zrt.
Acc.Number: 12010154-00175156-00100004
RÉSZVÉTELI FELTÉTELEK
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FIZETÉSI- ÉS LEMONDÁSI FELTÉTELEK
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Bankszámlaszám:
Kedvezményezett: Diamond Congress Kft.
Bank neve: Raiffeisen Bank Zrt.
Számlaszám: 12010154-00175156-00100004
Sponsors and Exhibitors
Arany támogatóink
Ezüst támogatóink
Exhibitors
Regarding the details of your appearance and needs you can contact the Congress organizer office in e-mail: ildikokruppa@diamond-congress.hu
Sponsors kit signup form