The future lines of research are: the improvement of the understanding of the biological mechanisms underlying neurodegeneration in AFD patients (deposition of Gb3 versus cerebrovascular lesions in the nigrostriatal network); the existence of a compensatory mechanism of increased a-GAL A activity upon PD neurodegeneration, as observed in LRRK2 carriers; the prospective multimodal comparison between AFD and sporadic PD patients; and finally the role of ERT chaperone therapy in neurodegeneration in AFD, remembering that ERT does not penetrate the blood–brain barrier. This evidence concerns the gene ELF3 and Nager acrofacial dysostosis.