Longitudinal effect of eteplirsen versus historical control on ambulation in <scp>D</scp> uchenne muscular dystrophy

R. Mendell, Jerry; Goemans, Nathalie; Lowes, Linda; N. Alfano, Lindsay; Berry, Katherine; Shao, James; M. Kaye, Edward; Mercuri, Eugenio

doi:10.1002/ana.24555

Public

Longitudinal effect of eteplirsen versus historical control on ambulation in D uchenne muscular dystrophy

Published in Annals of Neurology • Nov 17, 2015

Authors:

Jerry R. Mendell

Nathalie Goemans

Linda Lowes

Abstract

OBJECTIVE: To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with Duchenne muscular dystrophy (DMD). Three-year progression of eteplirsen-treated patients was compared to matched historical con...

Subject

Muscular dystrophy

Medicine

Internal medicine

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