New treatment opportunities in genetic neuromuscular disorders

ICNC2022 Symposia

Molecular treatments in genetic disorders is approaching. There are currently over 300 studies at various stages of translational efforts. Neuromuscular genetic disorders are on the top of the list. These studies are mostly being carried out in children. We aim to give a timely update in relation to clinical trials, followed by real life experience.

The principles yielded from clinical trials in pediatric neuromuscular disorders will have impact in other fields and topics such as hematology as already there is an approved gene therapy for hemophilia B. The discussion on molecular therapies to include gene transfer should now be regarded as ‘general culture’ in medicine. Physicians and other health care providers have been becomingly interested.


  1. To heighten how clinical trials in rare genetic disorders are designed and conducted.
  2. To understand emerging issues in real life experience
  3. To reflect whether the current status will match expectations
  4. To discuss possibilities and foresee the future

Symposium Talks

Establishing and maintaining an active site for clinical trials
Professor Yoram Nevo
Institute of Neurology,Schneider Children's Medical Center of Israel

Antisense oligonucleotides in Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA)
Professor Haluk Topaloglu
Yeditepe University, İstanbul

Gene therapies in neuromuscular disorders: pros and cons
Associate Professor Nicolas Chrestian
Centre Mère-Enfant-Soleil CHU de Québec

Emerging treatments in other genetic neuromuscular disorders
Professor Jiri Vajsar
Division of Neurology, Department of Pediatrics The Hospital for Sick Children