Tideglusib
Published , updated
Unassigned
New Medicines
Congenital myotonic dystrophy type 1 (CMD1) (Steinert disease)
Information
New molecular entity
AMO Pharma
AMO Pharma
Development and Regulatory status
Phase III Clinical Trials
None
Phase III Clinical Trials
Yes
Category
Glycogen synthase kinase 3 beta (GSK3ß) inhibitor; increased levels of GSK3ß reverse cognitive and behavioral deficits in transgenic models of syndromic autism.
Prevalence of DM in European populations is 10 per 100,000 people. According to the UK Myotonic Dystrophy patient registry, there were 556 patients with a confirmed diagnosis of DM1 registered between May 2012 and July 2016. Of these registered patients, 16 had CDM1 (classed as onset between birth to 1 month old [1].
Congenital myotonic dystrophy type 1 (CMD1) (Steinert disease)
Oral
Further information
Yes
To be confirmed