HTT and Huntington disease: CEP-1347 (500 nM) inhibited mutant huntingtin-associated neurotoxicity in an in vitro HD model, and CEP-1347 (s.c.; 0.5 mg/kg/day) reduced the decline in motor performance and restored cortical brain-derived neurotrophic factor BDNF levels in R6/2 transgenic mice of the HD model [38].