S1R agonists demonstrated neuroprotective effects in a variety of neurodegenerative disease models and are currently in clinical trials for a variety of neurological disorders including Alzheimer's, Huntington's, Parkinson's diseases and ALS (Brimson et al., 2020; Herrando-Grabulosa et al., 2021; Maurice and Goguadze, 2017; European Huntington's Disease Network et al., 2019; Ryskamp et al., 2019). This evidence concerns the gene TMBIM4 and glycogen storage disease VI.