TY - JOUR
T1 - Dollars and antisense for Duchenne muscular dystrophy
AU - Zingariello, Carla D.
AU - Kang, Peter B.
N1 - Publisher Copyright:
© 2018 American Academy of Neurology.
PY - 2018/6/12
Y1 - 2018/6/12
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85053891246&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85053891246&partnerID=8YFLogxK
U2 - 10.1212/WNL.0000000000005669
DO - 10.1212/WNL.0000000000005669
M3 - Review article
C2 - 29752302
AN - SCOPUS:85053891246
SN - 0028-3878
VL - 90
SP - 1091
EP - 1092
JO - Neurology
JF - Neurology
IS - 24
ER -