Tideglusib

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

Evidence based evaluations