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Quantification of dystrophin to assess the effects of dystrophin-restoring treatments in Duchenne muscular dystrophy: Lessons from the development of eteplirsen
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Quantification of dystrophin to assess the effects of dystrophin-restoring treatments in Duchenne muscular dystrophy: Lessons from the development of eteplirsen

Johannes Dworzak, Diane E Frank, Jay S Charleston, Frederick J Schnell, Jon Voss, Cas Donoghue, Anthony J Milici, Famke Aeffner, G. David Young, Steven A Moore, …
European journal of paediatric neurology, Vol.21, pp.e93-e94
06/2017
DOI: 10.1016/j.ejpn.2017.04.1209

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