This webinar will be delivered by Annemieke Aartsma-Rus, who is a Professor of Translational Genetics, Department of Human Genetics, Leiden University Medical Center in the Netherlands.
Joint webinar series by the European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN-EuroNMD) and the European Academy of Neurology (EAN).
Annemeike will briefly introduce the basic concepts of different genetic therapies (gene addition, genome editing, splicing modulation and transcript readthrough). Duchenne will be used as a paradigm since all these approaches are in development. However, the webinar will also take into account challenges and considerations for applying these approaches broader to other NMDs.
This webinar will take place at 14:00 (Paris time)
Further details about how to join this webinar will be available in due course.