Steroids: A Certain Can of Worms (A Complicated State of Affairs) - PPMD Community

Steroids: A Certain Can of Worms (A Complicated State of Affairs) - PPMD Community: Steroids are a complicated story and have clearly changed the natural history of Duchenne. We know they work for some, for most in fact, but not all. We know they increase strength and prolong ambulation, and statistics back this up. Predicting ambulation is prolonged on average for 2 years. But this is not the end of the story. Genetic modifiers have been identified that are thought to dramatically push out this timeline. Individuals with specific genetic modifiers continue walking into their mid- to late teens, often called outliers.