7th ERN EURO-NMD Annual Meeting presentations

Our annual meeting slides, focused on advancements in the neuromuscular field, are now live. Dive into the latest research findings and future plans.

Access the slides and let’s keep pushing boundaries in neuromuscular science!

Click directly on the session title to download the slides. 👇

7th ERN EURO-NMD Annual Meeting presentations

Session topic
Welcome note Teresinha Evangelista, LoĂŻc Carballido and Bertrand Fontaine
Presentation of ERN EURO-NMD activities

Moderated by Kevin Attal and Davide Pareyson

Education Teresinha Evangelista
CPMS Fernanda Fortunato
Guidelines Michelangelo Mancuso
EMQN scheme Carla d’Angelo
Update on the EURO-NMD working groups
News from the Patient Advisory Board François Lamy
Update on inter-ERN activities

Moderated by Nicol Voermans and Teresinha Evangelista

Gene therapy Kleopas Kleopa
Postgraduate Curriculum on NMDs Marianne de Visser
Mitochondrial diseases Michelangelo Mancuso
Channelopathies Robert Lauerer-Braun
Registry plenary session

Moderated by Hanns LochmĂĽller and Teresinha Evangelista

ERN HCPs participation in the Registry Paris team (APHP)
Registry structure and functioning platform Freiburg team (UKLFR)
Registry Hub and FAIRification Radboudumc team
Update on European projects

Moderated by Hanns LochmĂĽller and Shahram Attarian

ERICA Ana Rath (Orphanet – INSERM US14)
ERDERA/Rare Diseases Partnership Yanis Mimouni (EJP-RD)
Screen4Care Alessandra Ferlini (University of Ferrara)
Newborn screening session

Moderated by Susana Quijano-Roy and Ulrike Schara-Schmidt

Current and future perspectives on NBS Jim Bonham
Role of the NMDs community in NBS Manuela A. Santos
NBS for SMA, experience from Poland Maria Jędrzejowska (Medical University Warsaw)
The NBS workpackage in the Screen4care project Jan Kirschner
Patients perspective Marie-Christine Ouillade
Closing remarks

Satellite symposium

Present and future of Gene Therapy in Neuromuscular Diseases

Session topic 


Teresinha Evangelista (APHP, Paris)

Gene therapy and new avenues – Safety, limitations and new techniques

Francesco Saverio Tedesco (University College London)

Latest advances and perspectives for the development of gene therapies for NMDs 
Muscle diseases (DMD, LGMD, MTM1)

Serge Braun (AFM-Téléthon/Généthon, Paris)

Motor neuron diseases:

Spinal muscular atrophy

Jan Kirschner (University of Freiburg)

Amyotrophic lateral sclerosis

Vincenzo Silani (IRCCS Istituto Auxologico Italiano, Milan)

Charcot-Marie-Tooth disease and other neuropathies

Kleopas Kleopa (Cyprus Institute of Neurology and Genetics)

Accessibility to new therapies: the patients’ view

Alexandre MĂ©jat (EURORDIS Board member)

The ERNs are co-funded by the
European Union (Health Programme and CEF)

EU Commission

“EURO-NMD is one of the 24 European Reference Networks (ERNs) approved by the ERN Board of Member States. The ERNs are co-funded by the European Union (Health Programme and CEF).
For more information about the ERNs and the EU health strategy,
please visit ec.europa.eu/health/ern