Indeed, the inhibitor EX-527 displayed neuroprotection in Drosophila and in mouse models of HD, by improving motor function through inactivation of the FOXO3A and CREB pathways and the subsequent decrease in the expression of exon 1 of the Htt protein [72], and has been further studied in a few clinical trials (Phase I and II) [73,74]. The gene discussed is HTT; the disease is Huntington disease.