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. Author manuscript; available in PMC: 2015 Nov 1.
Published in final edited form as: Pediatr Neurol. 2014 Aug 7;51(5):607–618. doi: 10.1016/j.pediatrneurol.2014.08.002

Figure 2.

Figure 2

Effects of eteplirsen on dystrophin expression. (A) Representative example of time dependent increases in dystrophin-positive fibers throughout the study can be seen. Dystrophin expression begins between week 12 and week 24 of eteplirsen treatment, and continues to increase after that time point. (B) Western blot from a representative patient demonstrates measureable dystrophin at week 48 compared to baseline. (C) RT-PCR demonstrates presence of skipped mRNA product (289 bp) following eteplirsen treatment in the muscle of a representative patient with exons 49–50 deletion.