Beyond implications specific to neflamapimod, our findings inform on the impaired NGF-signaling based pathogenic model of cholinergic degeneration and the Ts2 DS mouse as a translational platform for target validation, for drug discovery and development, and for obtaining preclinical proof-of-concept support for therapeutic approaches to treating basal forebrain cholinergic dysfunction and degeneration associated with a range of neurologic disorders. The gene discussed is NGF; the disease is nervous system disorder.