Tiago and Philip at the Newcastle Centre with Dregan on his last day of the AMO-O2 trial.
Cure DM is pleased to be in close contact with AMO, bringing up to date information to the community, straight from the horses mouth. We often see so much infomration on line that we dont know what to believe, everything we publish here has come direct from AMO or has been double checked with them to ensure accuracy.
If you have any questions or queries, you can contact us (email@example.com) and we will put it to the team. Alternatively you can always contact AMO directly, via their website here: http://www.amo-pharma.com/amo_02.htm
AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has potential for use in additional CNS, neuromuscular and oncology indications.
AM0-02 is positioned to enter clinical stage development for the treatment of the severe form of congenital myotonic dystrophy known as DM1 or Steinert disease.
In cellular and animal models of DM1 and Duchenne muscular dystrophy, as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to increase. Inhibitors of GSK3ß have been shown to correct the activity of regulatory proteins, such as CUGBP1 in animal models of DM1. AMO-02 is an inhibitor of GSK3ß that has demonstrated pre-clinical efficacy in transgenic models and reversal of muscle cell deficits in ex vivo tissue samples in patients with DM1.
Inhibition of increased levels of GSK3ß is known to reverse cognitive and behavioral deficits in transgenic models of syndromic autism, a developmental disorder characterized by social communication deficits and repetitive behaviors.
After the positive findings from the recently completed trial, AMO is preparing to begin the next stage, which is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1)
We will keep you updated as we hear more via this page.