Article
Eteplirsen for the treatment of Duchenne muscular dystrophy.
Annals of neurology - 1 Nov 2013
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
Abstract excerpt
OBJECTIVE: 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). METHODS: DMD...
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