Willeke van Roon-Mom is full professor at the department of Human  Genetics at the LUMC and works on autosomal dominant neurodegenerative diseases that have aberrant protein aggregation as a pathological hallmark. She works in close collaboration with clinical departments as well as patient representatives, biotechnology and pharmaceutical companies. This research has resulted in 5 published patents, some licensed to companies as well as industry sponsored research projects. Originally her main expertise was in Huntington’s disease, a brain disorder caused by a polyglutamine expansion in the huntingtin protein. However, her group is now also focusing on other polyglutamine disorders as well as a very rare disease caused by a mutation in the APP gene called HCHWA-D (Hereditary Cerebral Hemorrhage With Amyloidosis-Dutch type) also known as D-CAA. For all these disorders she studies the molecular disease mechanism, identifies biomarkers and then uses this knowledge to develop novel therapies. She is co-director of the Dutch Center of RNA Therapeutics, founding member of the European 1 mutation 1 medicine network and scientific advisory board member of the N1C collaborative, that all that aim to develop antisense oligonucleotide based treatments for patients with ultra-rare hereditary brain disorders in The Netherlands, Europe and globally, respectively.