Dollars and antisense for Duchenne muscular dystrophy Eteplirsen and dystrophin

The world has changed for patients with Duchenne muscular dystrophy (DMD). Despite numerous important refinements in therapy over prior decades, a US Food and Drug Administration-approved therapy remained elusive until 2016. Now there are 2 such therapies, eteplirsen and deflazacort, in addition to prednisone, which has been used off-label for many years. However, controversies abound for both new treatments. Both are costly, and the pricing of deflazacort in particular has drawn criticism because it is a corticosteroid that has been in widespread use outside the United States for many years. © 2018 American Academy of Neurology.