EDG-5506 is a medication being studied for its long-term effects on safety, biomarkers, and functional measures in adults and adolescents with Becker Muscular Dystrophy. This study aims to understand how the medication can help manage symptoms and improve the quality of life for those affected by this condition.

Sevasemten is another medication involved in the trial, with the main goal of assessing its safety and tolerability when used over a long period. This medication is being evaluated to determine how well patients can handle it and to identify any potential side effects that may occur with extended use.

Becker Muscular Dystrophy – Becker Muscular Dystrophy is a genetic disorder characterized by progressive muscle weakness and wasting. It primarily affects the skeletal muscles, which are responsible for movement, and can also impact the heart muscle. Symptoms often begin in childhood or adolescence and may include difficulty walking, muscle cramps, and fatigue. As the disease progresses, individuals may experience increased difficulty with physical activities and may require mobility aids. The progression of muscle weakness is generally slower compared to other forms of muscular dystrophy, such as Duchenne Muscular Dystrophy. The condition is caused by mutations in the dystrophin gene, which leads to insufficient production of the dystrophin protein necessary for muscle function.