Tideglusib is being development for the treatment of congenital myotonic dystrophy type 1 (CMD1). CMD1 is a form of myotonic dystrophy type 1 (DM1), a rare, genetically determined neuromuscular disorder. CMD1 begins at or around the time of birth and is characterised by severe muscle weakness, cognitive impairment and other developmental abnormalities. The condition usually occurs when the mother already has DM1 and then it is passed on to her child in a more severe form. CMD1 is typically associated with significant medical morbidity and early death. No specific treatment is currently on offer, although supportive care to manage symptoms is available.
Tideglusib is expected to work by blocking the activity of an enzyme in the brain called glycogen synthase kinase 3beta (GSK3β). By blocking the activity of GSK-3β, tideglusib is expected to help reduce the severity of the disease symptoms. It is believed to promote cellular maturation and normalise abnormal molecular and behavioural characteristics. If licensed tideglusib taken orally, would offer a new treatment option for patients with CMD1 who currently have no effective therapies available.
rAAVrh74.MHCK7.micro-dystrophin is a medicinal product in clinical development for the treatment of children aged 3 months to 7 years with Duchenne muscular dystrophy (DMD). DMD is a rare progressive neuromuscular disorder caused by a gene mutation (change). DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. It affects …