Congenital Myotonic Dystrophy

Michael Snape, PhD, Chief Scientific Officer at AMO Pharma describes congenital myotonic dystrophy type 1 or Steinert disease.

Congenital myotonic dystrophy is a rare inherited disease in which babies have weak muscles and muscle tone – that affects their ability to move, talk, see, and breath. These children also have severe cognitive disabilities.

AMO pharma has clinical studies underway to investigate the safety and efficacy of tideglusib to treat children with congenital myotonic dystrophy type 1. Tideglusib is an inhibitor of glycogen synthase kinase 3 beta (GSK3ß), an enzyme that appears to be involved in the pathophysiology of congenital myotonic dystrophy.