AM0-02 is a clinical stage investigational medicine for the treatment of the most severe form of DM: congenital myotonic dystrophy.
In cellular and animal models of DM1 as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to increase. AMO-02 is an inhibitor that has been shown to normalise levels of GSK3ß in transgenic models and in ex vivo tissue samples in patients with DM1 and to reduce levels of the mRNA that is pathogenic for DM1.
Learn more about the current study: REACH CDM
Press releases and updates
September 15th, 2025: AMO Pharma Reports Long-Term Safety Data from REACHCDM-X Study of AMO-02 in Treatment of Congenital Myotonic Dystrophy Type 1