Willeke van Roon-Mom’s work is highly translational in nature, working in close collaboration with clinical departments and industry. 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. Her research is part of the LUMC research themes Neuroscience, Medical Genomics, Cell Tissue and Organs as well as Academic Pharma. The applicable NWA routes are regenerative medicine, personalized medicine, NeuroLabNL and health research, prevention and treatment.
Cure Rare Disease, a non-profit organization (Tax ID number 82-2473513), is qualified as a tax-exempt organization under section 501(c)(3) of the IRS and has been designated as a “public charity” under section 170.