AMO-04

AMO-04 is an investigational small molecule drug developed as a glutamate modulator, primarily targeting the treatment of Rett syndrome, a rare childhood neurodevelopmental disorder. The compound has shown significant promise in preclinical and early clinical studies, particularly based on screening by the Scout Program using mouse models of Rett syndrome. Its mechanism involves modulation of glutamatergic neurotransmission, which is believed to play a role in the pathophysiology of Rett syndrome. In addition to its primary indication for Rett syndrome, AMO-04 has also been investigated for potential benefit in certain breathing disorders and opiate-induced respiratory depression. The drug received Orphan Drug Designation from the US FDA for Rett syndrome and is classified as a new molecular entity[1][2][4][5][7][8].

Other names

AMO 04AMO04AMO-04