....from diagnosis to a cure!
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 information 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 (firstname.lastname@example.org) 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.
"This site is owned and operated by Cure DM, which is a registered charity. Nothing contained in this site is or should be considered, or used as a substitute for medical advice, diagnosis or treatment. The site owners and administrators cannot accept any legal or personal liability for the outcomes of actions taken by you in using this information. This site and its information do not constitute the practice of any medical, nursing, registered dietitian or nutritionist, or other professional health care advice, diagnosis or treatment.
All items and articles are written by individual authors. The opinions expressed are entirely the authors' own, except where clearly indicated. We strongly advise you to speak with a medical professional about all aspects of the condition."