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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 controls (HC).

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

Battini Roberta
2016-01-01

Abstract

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 controls (HC).
2016
Mendell, Jerry R; Goemans, Nathalie; Lowes, Linda P; Alfano, Lindsay N; Berry, Katherine; Shao, James; Kaye, Edward M; Mercuri, Eugenio; Battini, Roberta
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11568/950129
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