To date, few therapeutic approaches are available, but neurofilament light chain (NfL) and phosphorylated neurofilament heavy protein (pNfH) show great potential as biomarkers for neurodegenerative diseases in general, against which the progression of SCA3 in patients and in our knock-in (KI) model can also be well monitored [11]. Here, ATXN3 is linked to neurodegenerative disease.