At the moment, two compounds, the Sig1R and muscarinic receptor mixed agonist ANAVEXTM 2-73 (ANAVEX Life Sciences, ClinicalTrials.gov Identifier: NCT02244541) and the Sig1R antagonist E-52862 (ESTEVE, EudraCT number: 2012-000400-14) are being tested in clinical trials for the treatment of Alzheimer's disease and neuropathic pain, respectively, thus justifying the importance of Sig1R as a valid molecular target for clinical applications. This evidence concerns the gene SIGMAR1 and early-onset autosomal dominant Alzheimer disease.