…TIMESLOT: Tuesday 23 July 2024 – 15:00 – 16:00 CEST
…THEME: Genetic therapies and therapy developments for rare movement disorders
…PRESENTERS: Dr. Willeke van Roon-Mom (LUMC, The Netherlands)
Willeke van Roon-Mom is a full professor of Human Genetics, in particular of translational studies of neurodegenerative disorders. She studied Medical Biology at the Rijksuniversiteit in Groningen, and did her PhD in Auckland New Zealand studying Huntingtons disease. After a Post Doc in New Zealand, she returned to the Netherlands to work at the Human Genetics department at the Leiden University Medical Center where she started her own research group. Her work is highly translational in nature, working in close collaboration with clinical departments and industry. Unique patient-driven fund raising initiatives contribute not only financial input, but also patient perspective to research programs in her group. The main topic of her research is autosomal dominant neurodegenerative diseases that have aberrant protein aggregation as a pathological hallmark. She studies molecular disease mechanisms, identifies biomarkers and then uses this knowledge to develop novel therapies with a focus on RNA targeting antisense oligonucleotide therapies. She is the co-founder and co-lead of the Dutch Center for RNA Therapeutics that aims to develop RNA targeting therapies for patients with ultra-rare mutations.