Targeting Duchenne Muscular Dystrophy Targeting the Toughest Diseases

Before John Killian's toddler son was diagnosed with Duchenne muscular dystrophy (DMD), doctors told parents to simply take their children home and love them. The disease weakens the body's muscles and cognitive functions, which can lead to premature death. John’s son is now college-bound, thanks to the help of medical advancements that target the symptoms of the disease. Geneticist Dr. Alison McVie-Wylie and the research team at Vertex hope to move past only treating the symptoms of DMD by addressing the disease at its root cause.

Produced by Bloomberg Media Studios and Vertex Pharmaceuticals.