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- Title
Eteplirsen for the treatment of Duchenne muscular dystrophy.
- Authors
Mendell, Jerry R; Rodino-Klapac, Louise R; Sahenk, Zarife; Roush, Kandice; Bird, Loren; Lowes, Linda P; Alfano, Lindsay; Gomez, Ann Maria; Lewis, Sarah; Kota, Janaiah; Malik, Vinod; Shontz, Kim; Walker, Christopher M; Flanigan, Kevin M; Corridore, Marco; Kean, John R; Allen, Hugh D; Shilling, Chris; Melia, Kathleen R; Sazani, Peter; Saoud, Jay B; Kaye, Edward M; Eteplirsen Study Group
- Abstract
In prior open-label studies, eteplirsen, a phosphorodiamidate morpholino oligomer, enabled dystrophin production in Duchenne muscular dystrophy (DMD) with genetic mutations amenable to skipping exon 51. The present study used a double-blind placebo-controlled protocol to test eteplirsen's ability to induce dystrophin production and improve distance walked on the 6-minute walk test (6MWT).
- Publication
Annals of neurology, 2013, Vol 74, Issue 5, p637
- ISSN
1531-8249
- Publication type
Journal Article
- DOI
10.1002/ana.23982