Effect of Ataluren on dystrophin mutations.

Compared with DMSO control‐treated wild‐type siblings, a highly significant reduction in the relative amount of dmd transcript was detected in control‐treated dmd ta222a homozygotes (Figure 5 ), likely caused by non‐sense mediated decay of mutant dystrophin transcript. 31 However, the relative amount of dmd transcript in Ataluren‐treated dmd ta222a homozygotes compared with control‐treated homozygotes remained unchanged.

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