There are many open questions to be answered that will continue to rely on mouse models, including why mRNA levels are elevated, the importance of reduced FMRP in pathology, whether intranuclear inclusions are toxic or simply mark the progress of disease, and how protein sequestration and RAN translation contribute to the disease process in FPM and FXTAS. Here, FMR1 is linked to fragile X-associated tremor/ataxia syndrome.