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Eteplirsen treatment for Duchenne muscular dystrophy : Exon skipping and dystrophin production
Jay S. Charleston ,
Frederick J. Schnell ,
Johannes Dworzak ,
Cas Donoghue ,
Sarah Lewis ,
Lei Chen ,
G. David Young ,
Anthony J. Milici ,
Jon Voss ,
Uditha DeAlwis ,
Bruce Wentworth ,
Louise R. Rodino-Klapac ,
Zarife Sahenk ,
Diane Frank ,
Jerry R. Mendell
June 11 2018
May 11 2018
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Abstract
To describe the quantification of novel dystrophin production in patients with Duchenne
muscular dystrophy (DMD) after long-term treatment with eteplirsen.