LM22A-4 penetrates the blood–brain barrier reasonably well and has been shown to ameliorate symptoms of other neurological disorders associated with BDNF dysfunction including Rett syndrome, Huntington’s disease, Dravet syndrome, and refractory neonatal seizures (Canals et al., 2004; Chang et al., 2006; Gu et al., 2022; Kron et al., 2012; Ogier et al., 2007). Here, BDNF is linked to juvenile Huntington disease.