Although mutated TDP43 accounts for only approximately 4% of fALS cases (Figure 1), TDP43-SG accumulation is a pathology characteristic for ∼95% of all ALS and ∼50% of frontotemporal dementia (FTD) cases [21,55–57], as well as a secondary pathology in other neurodegenerative diseases, including Alzheimer’s [58], Parkinson’s [59] and Huntington’s diseases [60,61]. Here, TARDBP is linked to juvenile Huntington disease.