Looking ahead to the latest clinical trial results and new therapeutic options for neuromuscular disorders

Our team of writers are reporting live from Athens and have picked key notes to look out for from Day 1 - use the links to skip to the sections below, or scroll to view the full summary.

  • It’s All about Spinal Muscular Atrophy today! – learn about results from several ongoing clinical trials for SMA, what early diagnosis and treatment will mean for the future and what patients think about it
  • Gene and cell therapy – find out about current and new applications of gene therapy in neuromuscular disorders
  • Even more gene therapy – more details of Zolgensma® (onasemnogene abeparvovec) and what you can expect from it in clinical practice
  • EPNS Academy – learn about (auto)immune disorders in child neurology


The European Paediatric Neurology Society (EPNS) Congress 2019 will be starting rather sedately today with an opening session at 7 pm, giving everyone the chance to arrive and enjoy the beautiful weather in Athens. A pre-conference symposium on malformations of cortical development was optional during the day for members of the European Cooperation in Science and Technology (COST) Network on Brain Malformations (Neuro-MIG).

The current EPNS president Professor Sameer Zuberi together with Professor Dimitrios Zafeiriou, the president of the meetifng organisation committee, will welcome meeting participants. A highlight of the opening session will be the presentation of the EPNS Linda de Meirleir Award to Dr Hanna Hulshof from the University Medical Center Utrecht Netherlands for her outstanding abstract ‘Neuroimaging findings as biomarkers of epilepsy risk and neurodevelopment at two years in patients with Tuberous Sclerosis Complex (TSC)’.

A welcome reception in the Byzantine Museum Gardens will allow plenty of time to network and meet colleagues before the full scientific programme kicks in tomorrow. And what a packed programme it is.

It’s All About Spinal Muscular Atrophy today!

Early risers can take part in interactive teaching seminars every morning before the first plenary session. The subsequent parallel sessions on Wednesday are on neonatal neurology, epilepsy, neurogenetics as well as migraine. Epgonline.org will be discussing some talks from the neonatal neurology session.

We will then report from the ‘Time is motor neuron in SMA’ symposium which among others will feature interim results from the nusinersen (Spinraza®) phase 2 NURTURE (NCT02386553) trial in pre-symptomatic infants, a talk on accelerating time to diagnosis and the emerging challenges of the new SMA phenotypes. More on SMA straight after in Parallel session 2A with presentations on the long-term follow-up from the onasemnogene abeparvovec (Zolgensma®) phase 1/2a clinical trial NCT03421977) and on an interim analysis of the SHINE study in children with later onset spinal muscular atrophy (NCT02594124). Another symposium ‘Rewriting the tale of Sisyphus: the SMA boulder that went up a mountain and came down to change a disease’ will highlight the SMA patient journey through the eyes of a patient and a clinician. There will also be a discussion on the revolution in SMA treatment and what it will mean for the future. 

Gene and cell therapy

Fortunately, the third day of the EPNS Congress 2019 is somewhat lighter on the programme, at least for our therapeutic focus. One of the themes of the third conference day seems to be gene and cell therapy with a plenary session on gene therapy in the morning followed by several interesting presentations on the subject in parallel session 3B (Neurogenetics II). The ‘Gene therapy in Neuromuscular Disorders: the time is now’ symposium, sponsored by Avexis, will most likely mention SMA, but perhaps also something on their development programme on Rett syndrome and amyotrophic lateral sclerosis (ALS).

A dinner at the Island Club restaurant while overlooking the Athenian Riviera will allow for more networking and discussion with other conference participants.

Even more gene therapy

On Day four, there is even more on gene therapy – nearly all of parallel session 4B – Neuromuscular will be presentations on onasemnogene abeparvovec. Some of this looks like new data, so make sure you check back to find out. The Sarepta-sponsored symposium on ‘Development of therapies for Genetic Disorders’ fits right in – hopefully we will see some data on their Duchenne muscular dystrophy (DMD) gene therapy programme.

The afternoon Plenary session 6 on next generation sequencing (NGS) and beyond will be a nice change of subject. We will learn about the principles and practice of NGS, how it is used for disease gene identification in disorders with complex genetic architecture and if it will change daily clinical practice.

EPNS Academy: (Auto)Immune disorders in Child Neurology

On Saturday morning, the EPNS Academy on (Auto)Immune disorders in Child Neurology will teach us about molecular mechanisms that drive autoimmunity, autoimmune encephalitis, autoimmune disorders in children’s central nervous system, treatment approaches and testing strategies. A final session discussing the Highlights of the meeting will close out the conference.

There is lots and lots to see and do, check the daily blogs to have epgonline.org summarise it for you.

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Publications (4)
  • Even more on gene therapy…

    Even more on gene therapy today – see the new data from the SPR1NT and STR1VE-EU studies in spinal muscular atrophy and a thought-provoking discussion of the ethical dilemmas associated with gene therapy trials. What are the adverse events seen with gene therapy and how will next generation sequencing change your medical practice?

  • The last leg

    Discover the neuromuscular treatments that will be the talk of the EPNS Congress 2021.

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