P49 The efficacy and safety of Tideglusib in a randomized, placebo-controlled, double blind study in children and adolescents with congenital myotonic dystrophy (REACH CDM study)

Congenital Type 1 myotonic dystrophy (CDM1) is a severe, life-threatening disorder that affects approximately 1 in 40,000 children. It is caused by a genetic defect (i.e. excessive base pair repeats) in the region of the DMPK gene on chromosome 19. Affected youth experience a multitude of physiological problems, including muscle weakness, respiratory issues, gastrointestinal problems, and fatigue, while also experiencing cognitive challenges, speech and communication difficulties, as well as features of autism spectrum disorder.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research