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As part of the European Commission (EC) support for the functioning of the ERN registries within the current EU financial framework, EURO-NMD has allocated part of its budget to launch a pilot scheme aimed at supporting HCPs in populating the EURO-NMD Registry with patient data.The total budget allocated for the EURO-NMD Pilot Reimbursement Scheme is €123,000, corresponding to €1,500 for each participating HCP.The Data Entry Pilot Reimbursement Protocol outlines the process for reimbursing ERN EURO-NMD HCPs under this pilot scheme.
📌 Key conditions:
- 100 patients enrolled with mandatory EU RD data completed
- Cooperation contract signed with AP-HP (template available)
- Invoice submitted in line with contract terms
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EURO-NMD has conducted a large-scale survey to better understand the most burdensome symptoms reported by adults living with neuromuscular diseases (NMDs).
With input from 1,253 patients across the ERN, the study evaluated the severity of 28 symptoms using multi-criteria decision analysis (MCDA).
📌 What the survey showed:
- The most troubling symptoms were muscle fatigue, weakness, and reduced ability to move or stay active.
- Many also reported balance issues, muscle stiffness, mental tiredness, and pain.
- Differences were seen between disease types, showing that experiences can vary widely. The study highlights the urgent need for standardised patient-reported outcome measures (PROMs) to improve monitoring, guide research, and inform treatment decisions.
Capturing patient experience is essential for advancing truly patient-centred care in the field of NMDs.
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We are delighted to announce the second edition of the Summer School, co-organized by the European Reference Network for rare neuromuscular diseases (ERN EURO-NMD) and the French network of expertise on NMDs (FILNEMUS). Taking place from 24-27 September 2025, this Summer School will cover essential aspects of care, extending beyond the neuromuscular sphere. The program will include a wide range of topics to support healthcare professionals in addressing the diverse challenges of neuromuscular diseases (NMDs).
📌 Key topics:
- Extramuscular involvement: cardiac, endocrinological, gastrointestinal, skin and respiratory systems
- Dysautonomia, central nervous system disorders and epilepsies
- Rehabilitation
- Palliative care
- Psychological impact of NMDs
- Management of relationships and sexuality
- Management of pregnancy
Pre-registration is open until the end of May!
See you in Marseille ☀️!
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The 7th Neuromuscular Translational Training School organized by EURO-NMD and hosted by the Leiden University Medical Center (LUMC) focuses on therapy development from bench to bedside with a focus on neuromuscular disorders. The Neuromuscular Translational Training School contains lectures about the current state of the art of the management of acquired and genetic neuromuscular diseases, the different steps of drug development and the tools needed for this (outcome measures, biomarkers etc). It also stresses how patients should play a role in each of these steps and teaches how to communicate research to patients and the general public during interactive workshop sessions.
Registrations will open in early June!
📍 Leiden University Medical Center (LUMC)
📆 December 2-5, 2025
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EURO-NMD is pleased to endorse the 9th International Conference on Mitochondrial Disease and Novel Therapies, taking place 10–12 November 2025 at the Wellcome Genome Campus, UK, and online. This key event will bring together global experts in clinical and translational mitochondrial medicine to foster collaboration and accelerate the development of effective therapies. The programme includes keynote talks, abstract-selected presentations, poster sessions, and a panel discussion on lessons learned from clinical trials.
More information & a draft programme will be available soon! Endorsed by EURO-NMD, E-mit, and the Mitochondrial Medicine Society.
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All about FSHD - May 2025
EURO-NMD is excited to announce a 4-episode webinar series on Facioscapulohumeral Muscular Dystrophy (FSHD), coming this May. These free online sessions will cover the latest research and clinical developments in FSHD, with expert speakers from across Europe. Each webinar will focus on a key aspect of FSHD, including pathophysiology, clinical management, genetics, and emerging therapies. Participants will also have the chance to interact with professionals and gain insights from both scientific and patient perspectives. Don't miss this chance to stay up to date on FSHD research and advancements!
👉 Discover it today, the first two episodes are already available to watch!
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Next one, episode 3:
📅 Thursday 22 May 2025 | 🕓 16:00–17:00 CEST
Topic: Pediatric FSHD – Diagnosis and care for children
Speaker: Prof. Tracey Willis (Robert Jones and Agnes Hunt Orthopaedic and District Hospital NHS Trust, UK) This session will focus on how FSHD affects children, and how doctors can support young patients and their families.
Last episode, episode 4: 📅 Wednesday 28 May 2025 | 🕓 16:00–17:00 CEST
Topic: Clinical Trial Readiness for FSHD
Speakers: Dr. Nicol Voermans & Ria de Haas, PhD (Radboud University Nijmegen Medical Centre, the Netherlands)
Discover how researchers are preparing for clinical trials in FSHD and what patients need to know to take part.
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If you missed a webinar, don't panic! All our webinars are recorded and made available on our website. |
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Join Us at the European Human Genetics Conference in Milan – May 2025 Following successful events in 2014 and 2018, William Newman, President of the ESHG, invites us to Milan, Italy, for the next European Human Genetics Conference from May 24-27, 2025. Milan, known for its fashion, culture, and finance, will be buzzing with cutting-edge genomic discoveries. The conference program will include educational sessions, symposia, invited speakers, and posters, covering a wide range of topics in human genetics. Fellowship opportunities will also be available for younger members. The event will be hybrid, allowing virtual participation for those unable to attend in person. However, we encourage you to join us in Milan to experience the best science firsthand, build collaborations, and network with sponsors and exhibitors. Save the dates and get ready for an unforgettable experience in Milan! 📅 Dates: May 24-27, 2025
📍 Location: Milan, Italy Register for ESHG 2025 Hybrid Conference!
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The World Duchenne Organization is thrilled to announce that registrations are now open for the Duchenne Care Conference 2025. The online conference is taking place on May 26 & 27, 2025, from 15:00 to 18:00 CEST. It offers healthcare professionals and patient organization representatives a vital opportunity to stay informed on the latest breakthroughs and best practices in Duchenne muscular dystrophy (DMD) care. Last edition, the conference attracted 700 registrants from 72 countries. The Duchenne Care Conference is a unique platform aiming to connect healthcare providers and patient advocates with leading experts from around the world. Attendees will gain insight through a dynamic series of presentations, covering essential topics related to the care of individuals living with Duchenne and Becker muscular dystrophy. The Duchenne Care Conference is an annual online conference focused on Duchenne muscular dystrophy (DMD). DMD is a genetic disorder that causes progressive muscle weakness and degeneration. The conference brings together various stakeholders to discuss the latest advancements and innovations in the treatment and care of the disease. The conference also provides a forum for networking and collaboration among the Duchenne community.
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Join leading neurologists, researchers, and healthcare professionals in Helsinki, Finland, for the 11th Congress of the European Academy of Neurology (EAN). This premier event will feature pioneering research, plenary lectures, and interactive workshops, covering a wide range of topics, including neurodegenerative diseases, stroke, epilepsy, and multiple sclerosis. Helsinki’s vibrant culture and stunning surroundings provide the perfect backdrop for networking and knowledge exchange. Don’t miss this essential event for advancing neurology and improving patient care.
Submit your membership applications before 31 May for reduced rates. Don't miss out on this opportunity to connect, learn, and save!
👉 Register for the congress here:
https://ow.ly/NeMS50VaQPI
📅 Dates: June 21–24, 2025
📍 Location: Helsinki, Finland Stay tuned for registration details on the official EAN Congress website!
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As part of the ENTRY-DM European research network, which will recruit 14 full-time PhD candidates across Europe, a position is now open in France focused on decision-making in DM1 patients and their participation in clinical trials. The ENTRY-DM programme aims to advance antisense oligonucleotide (ASO) therapies for Myotonic Dystrophy through cutting-edge, collaborative research.
Candidate profile:
- Master’s degree in neuropsychology, psychological sciences, or neurosciences
- Proficiency in English and French (due to patient-facing work)
- Must not have lived in France for more than 12 months in the last 3 years This is a unique chance to contribute to translational research within an international training network. |
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Stay updated with AFM-Telethon's "Advances in Research" documents AFM-Telethon's "Advances in Research" documents, updated annually in June, provide a comprehensive review of the latest medical and scientific discoveries in neuromuscular diseases. These valuable resources help keep the global neuromuscular community informed about the latest research developments. Highly respected within the field, many of these French-language documents have been translated into English for wider accessibility. Additionally, the CMT Advances are also available in Italian, thanks to the support of the Italian association. Additionally, the "Overview of Neuromuscular Diseases" by AFM-Telethon provides a concise description of all neuromuscular disorders, including information on genes involved, ORPHA and OMIM codes, and treatment recommendations. This handy pocket guide offers a quick reference for the entire field. |
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