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- W1502517079 endingPage "426" @default.
- W1502517079 startingPage "417" @default.
- W1502517079 abstract "Targeted dystrophin exon removal is a promising therapy for Duchenne muscular dystrophy (DMD); however, dystrophin expression in some reports is not supported by the associated data. As in the account of 'The Emperor's New Clothes', the validity of such claims must be questioned, with critical re-evaluation of available data. Is it appropriate to report clinical benefit and induction of dystrophin as dose dependent when the baseline is unclear? The inability to induce meaningful levels of dystrophin does not mean that dystrophin expression as an end point is irrelevant, nor that induced exon skipping as a strategy is flawed, but demands that drug safety and efficacy, and study parameters be addressed, rather than questioning the strategy or the validity of dystrophin as a biomarker." @default.
- W1502517079 created "2016-06-24" @default.
- W1502517079 creator A5001672241 @default.
- W1502517079 creator A5054694810 @default.
- W1502517079 creator A5080459494 @default.
- W1502517079 date "2015-07-01" @default.
- W1502517079 modified "2023-09-25" @default.
- W1502517079 title "The emperor's new dystrophin: finding sense in the noise" @default.
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