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.
Bibliographical noteFunding Information:
C. Zingariello reports no disclosures. P. Kang has served on the editorial boards of Pediatric Neurology, Pediatrics, Neurology, and Muscle & Nerve; holds a grant from the National Institute for Neurologic Disorders and Stroke of the NIH; performs pediatric electromyography (5% effort, 2014–2018); consults for AveXis and Fondazione Cariplo; is a coinvestigator for Catabasis; has received research support from Catabasis Pharmaceuticals, Pfizer, and Genzyme; receives royalties from Springer for a textbook; serves as an associate editor for Muscle & Nerve and thus receives honoraria from Wiley; has written chapters for UpToDate and thus receives honoraria from Wolters Kluwer; previously served as a site principal investigator for a clinical trial sponsored by Pfizer; previously served on the DMD Advisory Board for Sarepta Therapeutics; has received travel funding/speaker honoraria from the American Academy of Neurology, American Academy of Pediatrics, Child Neurology Society, Amy & Friends Cockayne syndrome network, the University of California San Diego, Florida Hospital, and Paediatric EMG Congress and previously served as a coinvestigator for a clinical trial sponsored by Sarepta Therapeutics; his spouse holds a patent for compositions and methods for treating glycogen storage diseases (08730667.6-1212 PCT/US2008054911, filed February 25, 2008 and has thus received royalty payments from the University of Florida Research Foundation. Go to Neurology.org/ N for full disclosures.
© 2018 American Academy of Neurology.