论文部分内容阅读
Eteplirsen induces skipping of exon 51 in dystrophin pre-mRNA in the muscles of DMD patients treated with the drug.This treatment restores the reading frame of the defective dystrophin mRNA, leading to translation of functional dystrophin protein.Efficacy of eteplirsen was demonstrated in key clinical measures such as six-minute walk test and pulmonary function test.Importantly, the drug was well tolerated throughout the trial and there were no significant treatment-related adverse events.The results of the continuing trial after 120 weeks of treatment will be presented.