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Article

Eteplirsen treatment for Duchenne muscular dystrophy

11 May 2018

Abstract excerpt

Objective To describe the quantification of novel dystrophin production in patients with Duchenne muscular dystrophy (DMD) after long-term treatment with eteplirsen. Methods Clinical study 202 was an observational, open-label extension of the randomized, controlled study 201 assessing the safety and efficacy of eteplirsen in patients with DMD with a confirmed mutation in the DMD gene amenable to correction by...

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PMID: 29752304
DOI: 10.1212/wnl.0000000000005680

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