The Race to Yes – we need you to sign
Duchenne is the leading genetic killer of children. The disease affects one out of every 3,500 boys born worldwide. With no approved treatment, Duchenne has a 100 percent fatality rate. A child of five diagnosed with Duchenne experiences a loss of muscle strength leading to wheelchair confinement by adolescence and a shortened life assisted by ventilators. The progressive deterioration of muscle strength leads to death in the late teens or early twenties.
Today, leading scientists see a decisive decade for Duchenne research. The success of landmark clinical trials for the breakthrough drug eteplirsen is a pivotal moment that will lead to the development of additional new treatments for this dread disease. A pending FDA decision could determine whether this generation of children is the last to die from Duchenne, or the first generation to live.
This infographic charts what happens when urgent need, sound science and good policy combine to help FDA decision makers say “YES” to safe, effective treatments for Duchenne.